Success Reveal
Robert Kiyosaki
How to become a millinaire in 3 Years or Less
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All right, so we're moving into the second part of our discussion
Which is about current and future approaches to treating
PSC in lamp so we're going to build on you know you've described to us the symptoms and burdens that
affect your daily lives and now we want to hear about
How you manage those whether that be through drug treatment or other?
Types of interventions or even lifestyle modifications to start our conversation we have our second panel
So we have Nikki Lindsey Nicole and Madeline
our first speaker
Nikki
Unfortunately it was not able to be with us today
She's at EsC and Lam patient who actually received a lung transplant just a few months ago
And due to recovery she was unable to be here
But her desire to have her story shared
Despite going through the recovery. I think is a testimony to this community's commitment and tenacity
And I believe she actually is on the webcast tuned in today, so I'm happy to have her share by
audio recording her her testimony with you I
Attended Tuber sclerosis complex since birth I had started having a hotel thousands at 7 months of age
Lucky a specialist in Minnesota organized the symptoms and diagnosed me when I was 3 I
Have not had an active seizure since then in the moment since the age of 5 but I do live in fear than returning I
Was diagnosed with when I'm at 26 at that time rapidly and was not yet a treatment
I had about 6 months of normal lung function after diagnosis
When I was offered the drug my lung function was about 47%
They helped my quality of life improved my decline, but it did not prevent me from meaning oxygen or a double lung transplant
I am hoping however since my kidneys have responded
Well that it might prevent me from eating kidney transplant in the future however. There is no guarantee
Wrap immune had mixed results in my lungs my decline was not fast is when I was off of it
But I still had drops I felt better for a long time on it even when the time for Oxygen keen I could
My body handles being on the drugs better than off once my airways of healed my transplant team book will be back on
While rare it has been known that gland can regrow but can regrow after transplant
My life pre transplant and post transplant is difficult well
I no longer need a tech leaders of oxygen in the function like I did prior to
Transplant its steroid induced diabetes for about five months after I
Still have to remain vigilant of my blood sugar's even though they have improved my energy levels and stamina are so unpredictable
They also end up using a lot of reminder apps and worms because I'm more forgetful
I have to be very vigilant being aggressive even with minor illnesses like allergies because of the IMmuno suppression
My body has no immunity now to avoid rejecting my lungs some days
I am unable to exercise due to nausea and/or pee the drugs are hard on my kidneys period As the way myself take my temperature
Blood pressure and lung function measurements on a daily basis for the rest of my life. I need periodiC Gronkowski's to check for rejection
My best weapon on building one function despite living on a measly 12 percent of my lungs prior Transplant was exercise
I continued my pulmonary rehab type program at a local hospital Fitness Center on my own
When I was exercising on my own at first I was also part of an exercise study through the land foundation
I see a therapist and psych nurse practitioners to be sure I am monitoring my anxiety it seems from all the issues that
TSDm land has claws even before transplant. I was hardwired to stress I
Had to stop working in 2015 I
Learn how to shift to raise the money for transplant in order to keep my insurance and cover unexpected
Cost as well as pay deductible expenses
I don't know if I will be able to return to work, but being on disability as a whole process is very hard
But I do know that working became impossible pretty transparent I
Also, sacrifice as much time with appointment with appointments and transplant aftercare the first six months I've had appointments at
Least every two to three weeks, or once a month of things go well
My ideal treatment would be a drug or combination
before the Growth
But also kill the tumors what they be in the kidneys or lungs or both
It would also ideally be a cocktail that would not have to be taken daily
perhaps only for periodic bursts that have lasting effects
I would have the minimum aggravating side-effects in an ideal world for example
No, nausea diarrhea or stomach pain, or at worst case a tolerable amount like rapid mean
When I first slide about the new treatment
I weighted against the worst case outcome our interaction would look like
Would it make me worse off in the case of wrapping you know the fourth or risk to me because I could not get much
Worse than I was at the time I felt better also using a known drug rather than an untested one
At least there was long term data and kidney transplant patients of what the effects long term use
Would have this was another thing I weighed in any new drug scenarios
to be honest with the sick as I was try to rapping in I might not have qualified for a lung transplant because of my
Kidneys and the way the tumors there were affecting their function. I also had at least two brushes of death
Turning 40 was looking way out of reach. I exceeded those past life expectancies Lam, but not that much
Now being post transplant. I know I am living in overtime, so to speak
But it was the only option I had left other than death
This disease does word are actively trying to kill me and take my life
I
Was fortunate enough to at least have life preservers at the exact moments and needed them both wrapping in and transplant
But neither one are care. They are merely a treatment and treatment not everyone responds tolerates or has access to I
Think if research has shown other pathways other than enter
Involved we need to expand our drug screening and agent selections to those alternative pathways as well in the hopes of learning more
But also potentially helping other people
Especially for those who don't seem to respond and tour inhibitors
Good afternoon
My name is Lindsay Goldman, and I'm 34 years old. I was diagnosed with Tuberous sclerosis complex at age 5 and Lam at age 21
When my parents were told in 1988 that the small red bumpy spots on my face were signs of a disease called t Sc
The doctors opened a book and painted the worst possible future for me in the eighth grade
I was very active in sports and the angio fibromas on my face began to bleed during exercise
After seeing a dermatologist my family and I decided to try a procedure
Called Dermabrasion that would chemically peel off the first several layers of my face and actually shave off the angio fibromas
It was the first of several treatments. I tried, but was unsuccessful
Besides the angio's on my face. I was asymptomatic until the age of 17
I just got done setting a national record in the 2000 meter relay at the national inline speed skating
championships and had an opportunity to visit the Olympic training Center, I
failed my physical I
Was on the operating table for ten hours while my urologist performed a partial nephrectomy in attempt to remove over 20 large
Aml's from my kidneys
Over the next three years I had about five kidney embolization procedures to maintain and/or Shrink the aMl's
To my knowledge surgery was the only option at that time I
Was running one day when I suddenly felt a sharp stabbing in my chest it?
Hurt to breathe and to stand up straight. I
Suffered through the pain for about three days when I decided to go to the er after
Some tests the doctor immediately told me to sign some papers, so they could insert a chest Tube
He said your less long as 100% collapsed and is pushing on your heart and trachea. We have to put a Tube in right away
That was my first experience with land at the age of 21
Over the next Few years. I averaged about three lung collapses a year which cut my college volleyball career short I
Had a pleurodesis performed on both lungs a couple of times, but it was only a temporary fix
During the same time. I was again struggling with the kidney tumors
I have one that is wrapped around her the may artery's on my right kidney
I am grateful to have wonderful physicians in the Houston medical Center however
I had to drive to Cleveland clinic several times over the next year to have doctors. They're trying to embolize my tumors
Every physician that treated me always said there weren't any drug treatments from my amls or my lamb
In 2009 I spent 21 consecutive days in the hospital. Why doctors tried to figure out how to get my left lung to re-inflate?
Since the chest Tube and Pleura Deesis wasn't working. It was day 22 when my right lung collapsed
My pulmonologist came in and said we were transferring you by ambulance downtown to the medical center
We don't know what else to do for you
The next 25 days were the hardest days of my life
I decided to have a bilateral pleural me a
Procedure that removes the pleural lining or the protective lining around my lungs and glues the lungs to my chest cavity
The left was done first and the right was done ten days later. I had six chest tubes in at one time
During my recovery. I was referred to an oncologist that specializes in in tour inhibitors
I was immediately prescribed wrap immune and was instructed to take five milligrams each day I
Suffered through many of the typical side effects such as mouth sores awful Gastro issues and hormonal changes
however the tumors on my kidneys liver spleen and face began to Shrink I
had improvement for about three years until my doctors suggest that I switched to a finit or I
Have been on a spinet or for almost four years and tolerated much better than the rapp immune
The affinity is more for maintenance
It isn't shrinking tumors like the rapp immune did but it is keeping me stabilized
I have had small pneumos, but I haven't had a chest tube since 2009
After listening to my rollercoaster ride of the story. I would like to express my concern for future treatments
Making the decision to have a bilateral or rectum
II was a tough one however hearing that it may be nearly impossible to remove my lungs for transplant one day is heartbreaking I
Already know from my decline that a one transplant is necessary, but what other options do I have if doctors can't remove my lungs
I'm extremely grateful for the drugs that are available now, but what about my future?
My doctor explained to me that
Mtor inhibitors aren't made for long-term use and that there's a chance my body will eventually find a way around it
So what do I do after the offense or stops working? I dream about a treatment that would improve my breathing and
Help minimize the cyst on the lungs yes the indoor inhibitors have stopped the progression of my diseases
But I wish my quality of life would increase I have taken part in exercise studies and trials for researchers to learn about my diseases
I'm currently an elementary physical education teacher that fills the effects of my lamp every day
Even though my lungs are stable I can no longer
Explain and demonstrate an activity to my students because I get too out of breath
I
feel my teaching career will end sooner than I want it to I want to be able to run around with my
One-Year-old daughter and watch her grow years from now. We were blessed to adopt Maddie when she was 3 days old
I was relying on her
I am relying on the FDA and other specialists to create future treatments to fight TSC and liam
I have tSC and lan but gia seen lamb doesn't have me thank you for your time
Good afternoon
I'm Nicole with the sporadic lamb patient from Michigan I
Am the mother of a small child a wife to a great husband a daughter a sister and a friend
I'm an attorney with a thriving law practice and a consultant that travels a lot
I tell you these things because these facts all framed my mental and emotional response to treatment and
how I approach both the long and short term view of managing lamb I
First started experiencing serious symptoms of lamb about six months prior to my diagnosis although with what I know now
I believe I had symptoms even before that
like many patients
I was not taken seriously by my medical doctor and many of my concerns about my symptoms were brushed off or ignored
His opinion was simply that I had anxiety and nothing more I
knew my body and I knew he was wrong, so even though by Christmas Eve of
2014 I knew I needed to go to the hospital my self-doubt made me wait until jan you for 2015
And I still refuse to see that Dr.
Lam had progressed to the point that I was finally unable to carry out any day-To-day activities and
Like many Lam patients, I appeared those on that day to be completely healthy almost exactly as you see me today
At that point. I didn't have the energy to get up off the couch much less
Take care of my then four-year-old son my husband forced me to go to an urgent care
once I was there an x-Ray clearly showed a large amount of fluid in my lungs I
cried not because I was scared, but with relief
Despite what my doctor said I wasn't crazy I?
Went straight to the emergency room at the hospital and had one point two liters of Kyle drained from my pleural cavity
From then I almost spent I spent almost 30 days in the hospital
Because my lymphatics were draining Kyle into my chest and the leak would not resolve it was so bad
I was secreting up to three Lua 3 litres of fluid into my chest cavity a day and
my doctors were worried that my body would eventually give up as a
Result I had three surgeries
Was pressured to have a fourth which I refused
Was placed on a fat-free diet and was taking multiple drugs?
Even though I was at one of the premier hospitals in the country and one that does specifically treat, Lam
I was not given in my opinion the right advice about how to treat or manage my condition
as
a result of my own research and strenuous self-Advocacy to my medical doctors
I was discharged from the hospital with a semi-permanent chest tube to continue the draining and was taking two milligrams of sur almost daily
Over the course of about two months the Kyle leaks slowly subsided
My body responded very well to the sera lomas, and I was able to have the chest - removed in
This respect the drug has been a literal miracle for me
Because all previous surgical nutritional and drug interventions were not able to stop my repeated Kyle, Thorax
from April 2015 to January 2017 I would say that I had a very good experience with the drunk and
Supplemented my care and healing with yoga which I believe also had a tremendous positive effect
However over the last several months. I've had serious side effects related to taking sir ominous including headaches and major gastrointestinal pain
I've attempted to modify how I take the drug including skipping days and taking lower doses more times throughout the days
However none of these have been a satisfactory resolution
Because I notice a negative difference in my breathing as a result
I'm now at a point where my question is which type of crappy do I want to feel on a day-To-day basis
My reality today is that even though the only prescribed drug. I take is surah mmUs
I have no other drug options to treat or mentor to my disease
this is important because like all patients with lung disease one of the biggest issues with treatment is being unable to breathe and
the uncertainty of tomorrow
Many of us are considered by the medical community be highly anxious?
people and while that may be true on some level one of the treatment factors not taken properly into
consideration is the mere fact and when you cannot breathe or
When you don't know what betrayal your body will do it on you next there is an automatic panic response
For many of us the memory of this does not go away easily. I consider it to be like pTSd
I see it myself, and I see it in so many other patients
So in considering treatment for Lam and other lung diseases
I'd love to see treatments that not only have lower side effects
But also treatments that address the mental and emotional aspects of being able unable to breathe properly and coping with a chronic health condition
My whole goal now is to be able to manage the side effects of strongness
Because even though the side effects have increased over time
I have no other treatment option available to me at this time of my Kyle return
This is a life-or-death choice as far as I'm concerned
additionally I do not want to lose any one function, and I know that this is my best chance of preserving it I
Lead a very active life including a large amount of travel for business
And I don't want to change any of that or allow my diagnosis to dictate how I live day to day
unlike many other patients
I've almost always been able to realize that goal that
Are not confident that if this drug stops working for me, and I have to stop taking it that it will remain that way
Thank you for listening
Good afternoon. My name is Madeline Noland I
Want to thank you for this opportunity to share my experiences with sporadic Liam
I was diagnosed in 1999 at the age of 47 and since then have only had lung involvement
to deal with my journey with Lam
For years there were no treatment options suitable for me the treatment options back in
1999 involved Hormone treatment
Because liam was a woman's disease
It had to be our hormones
There were no studies at that time to support this treatment
so instead of
Hormone treatment. I did what I could just just support my overall health
My goal was to support my body. So would have only one battle I
participated in research at Nih with visits once or twice a year from 1999 to
2015 I
actively participate with the Lam foundation as a former board member and the new England lays on for patient support as
My lung function particularly my diffusion rate to exchange gases it steadily declined over the years
from 60% down to 40%
it was then I decided to take cyril imus hoping that it would slow down the destruction of my lung tissue I
Carefully considered the possible side effects and in 2011 I took my first drug to treat lan
after several years of more stable lung function I again began to decline significantly
Now I'm near 30%
My physicians, and I tried increasing the dose of sur alamos hoping to put the brakes on the disease progression once again
Then however didn't help in
Fact it brought on other problems of severe edema in my legs, and it did not help my lungs at all
I
Had to seek out physical therapy
Treatments for the Lymphedema in my legs it took months for my right leg to find its way back to almost normal
even after
Discontinuing the higher dose of sur alamos. It was as if the Dam had broken and it could not be completely repaired
Until that time the side-effects weren't much of a problem
Minor things like skin breakouts some little stomach issues. They were all worth it for breathing better
so with progressively declining lung function my physicians recommended that I begin evaluation for lung transplant as
of may
2016 I am listed for a double lung transplant at Brigham, and women's hospital in Boston I
Continue to take sir alice as I await the call for transplant. It isn't clear if sir alamos is helping anymore
However, it's very possible a more rapid decline could happen if I stop taking it all together
Since 1999 I've been using supplemental oxygen at first I needed it only for exertion two to four liters
Then I added it for sleep
Now you need it 24/7 and use over 60 liters for exercise
Supplemental Oxygen is not a treatment for Lam, but it allows you
Patients and me to lead in an adjusted active life I can breathe enough supplemental oxygen
to continue to teach at a public high school
Reluctantly I had to give up teaching physical education and now just health classes
The challenges over the years of using supplemental oxygen have been many
Finding the right equipment for my active life took some time
Teaching in a gym everyday was a challenge with a metal oxygen tank in the back
It just didn't work that well
Thanks to networking with other limb patients. I learned about liquid oxygen
It was a godsend for me to maintain a quality of life
I could work all day without needing to haul in tanks and change them frequently
Sadly many patients have had their liquid oxygen systems taken away due to changes in insurance coverages
This is another important issue needing attention
the success of current treatment of sur alamos to slow down lung damage varies from patient to patient
Some questions that patients want answers to her when should you begin?
Treatment what dose is effective for you? How long is it safe to take it and
Besides PFds, how do we know if it's working?
The scientific community researching Lam work so hard to find answers to these and many other questions
They have many ideas or other possible treatments
Finding a treatment that stops limb completely not just slow it down is the goal
Lam patients although few in number were active participants in research
We're educated about our disease and with the help of the Lam foundation and the Lam clinic Physicians and staff
We know we know a lot about Lam
For me to damage to my lungs is severe the lungs are full of holes
What I really need is a treatment that can regenerate the lost lung tissue so that I can avoid a transplant
Am I dreaming?
Well Perhaps this won't happen in my lifetime
But in the lifetime of newly diagnosed young women this could be a reality
Thank you
Thanks to each of you into to Nikki if you're tuned in thank you also for your testimony
will now move into our
Second and final set of polling questions related to current treatment options
so the question here is
Which drugs for tse and lamb have you or the patient that you care for?
Tried and this question will be as in the form of select all that you that apply
So the options are one rapamycin or sear align this to ever align this
3 by Gaba Trend for Diazepam
5
Acth 6 Cbd
Seven other Cedar drugs 8 doxycycline
9 steroids or 10 some drug. That's not listed here
you
As of these treatments that you or your child have tried again select all that apply
One no, positive benefit or benefit was lost over time to you weight gain or weight Loss
3 a compromised immune system for mouth sores
5 constipation 6
inability to enjoy activities that were once enjoyable 7 vision issues or vision Loss 8 nausea diarrhea
9 pain or 10 some other downside that's not listed
you
Okay, so the most common
Downside that you've reported
Or that you either had no positive benefit from using the drug or that benefit was lost over time
then the next highest was other some other reason so we'll
Certainly want you all to raise that during our discussion
There was also a fair amount of experience with weight gain or weight loss compromised immune system
As well as the inability to join activities that were once enjoyable
Then there was also
experience with all of the other downsides with the
Only a couple of you
having issues with vision
alright, so
Now that leaves us with our final
moderated audience discussion of the day
So again, we're focused on current approaches to treatment
Current and future approaches to treatment our questions for our discussion are what are you currently doing to help treat the condition or its symptoms?
Again, I'll reiterate this is in addition. Not just drug products, but also
Other things that you're doing to help treat or manage the condition or its symptoms
also, how well
Does your or your the patient that you care for is current treatment regimen treat the most significant symptoms of the condition?
What are the most significant downsides?
to the current treatment and how do they affect your daily life and
Then kind of switching gears and thinking about the future treatment. We want to know sort of a cure
what specific things would you look for in an ideal treatment and
What factors to you or the patient you care for take into account when making decisions about?
selecting a course of treatment
All right
So we we heard a lot of discussion already about?
Carolinas, but I was wondering if those in the audience had either similar or different experiences with
that product
and if you could describe
not only any benefit, but also any potential loss of benefit or
adverse events that may have impacted your decision to use it over time or is there anyone that has considered using it and
for any reason decided to
not
use the product
So I mean any takers count Calvin I fear you have comments from the web all right
So we have a comment from the web from Scott Moskowitz who has had vision issues that were
treated with a
Senator so he says quote
I have a retinal astrocyte Econo Toma which hemorrhaged three years ago as well as two sub ependymal nodules and
Angioma lipoma skin manifestations and partial onset seizure disorder which is just diagnosed at the time?
I went to every expert at the last world TSC
Conference to find out if the finit or would work for shrinking the tumor in his eye and they said it should work
But there was no study or paper on it, and it was and it would be considered off list
I am thankful that the hemorrhage dried up after 14 months and when it reoccurred it dried up after 6 3 to 6 months
Thankfully I didn't have to make hard decisions about losing my eye
Thank you
So I was just throwing out one drug product they started off, but we can open it up
That was a great example for a senator
just a reminder state your name and
Also, if you're affected with PSC and or land where state your site, this is Mary
and I have lamb I am taking rapa and I've had to go on increasingly higher and higher doses and
moderate lambs
so the progression over time it's been very slow and
but there's definitely progression and
I've had high blood pressure issues with it treatable, but high blood pressure with it. So my question is how
Much longer will we really be able to say maybe helping me
My progression has really
Changed when you look at it
or
Will I need something else and will that be available?
other approaches to treating the burdens that we discussed in Topic 1
there's a lot of we had a lot of drug examples, but it sounds like
Certainly lung transplant is something that was discussed about a lot by our panel. So if you have experiences either
Potentially needing the lung transplant or having gone
Experience the lung transplant to be interested in hearing about that and there also was quite a few other
responses other drugs that have been tried and
Other downsides of drugs that have been tried, so it'd be very interested in hearing about what those experiences were
My name is Karen Kinsey, and I asked faradic Lam
I've been on rapa tour
Probably six years and at first like I said before
Increase my FEV1
Roughly 10% which I was ecstatic about and some of the common side effects
I mean, there's two pages of side effects when you get the drug from your pharmacist, but include high blood pressure high cholesterol
Edema I
In addition to some other land women I know have had
Constant very heavy periods
At One point I had
Solid period for eight weeks, and then stopped for two weeks and six weeks, then stopped in seven weeks
So I ended up having an ablation
to stop that and
My gynecologist said you're never going to have a period again here. Don't worry about it
And it may be because I'm menopausal age. I don't know but the fact that other land women had the same problem
I've had had the same problem. I find that there is likely a connection
But I still think it's worth it's worth all of that
To have that job and when you described you said when you first started taking the drug you
I think piece of your FPV score went up 10% on Major estatic
Where did you notice things that you're able to do?
From my status. I was less fatigued
and I I
Was happier because I could move around a little bit better, so yeah, definitely 3 thank you
their experiences purchased the treatment
You have a comment
Hi, I'm deaf guess. I have tse, Amylin and
So I recently started taking rapa about
Probably about two and a half months ago now
but it was actually for a combination of both conditions because my aml's are so large and
I have about seven in validations
and I'm to the point where symbolization czar no longer an option and so it's kind of interesting because
I don't quite yet know if the rap is
Effective on my lungs based on the time that I've been taking it but very interested to see if that's going to be helpful
Also, really interested to see if it helps shrink the aml's that I do have already existing
Since it was a fairly recent decision for you to go on drugs you tell us
About how your decision tell us about your decision making
You know so you know we've heard about the list of side effects that have you know come with that product
Yeah, how did you weigh those with the potential benefit and what made it?
You said the embolization you that's no longer an option. What else made it now the right time
To consider that treatment. Yeah, it was really a lack of other options um I
Had an embolization that went pretty awry and lost a lot of my kidney and so between my two. I barely have one
and with the aml's really impeding on them both I
couldn't chance
Been getting any more aggressive so had to do something to stop it
Perfect. Thank you
We have a comment in the back I
Just wanted to highlight
Something that has come up today about this sub population the 5% of people with clear sea that have polycystic kidney disease
That was a story of my my sister, and she passed away seven years ago
And I know frank mentioned it today that the current approach to treatment is supportive care
And I think as the other aspects of Tuber sclerosis, and I mean she had lamb as well improve
I think we're sort of left to kind of maybe the low-hanging fruit we've picked and maybe it's time to
Start looking at that 5% which I think my back-of-the-envelope
Calculations is a thousand people in the Usa and have a look at you know what?
We could do because the supportive care itself is not necessarily helpful as highlighted kidney transplant is often not possible because of other
health conditions associated with these chronic diseases
Dialysis was not an option because of profound intellectual disability and challenging behaviors in my sister's case
And I think that's something that we should look at and look for better treatments
Thank you
other
Experiences with your current approaches to treatment again does not need necessarily be
Drug therapy oh we have Madeline and then nicole
So I
try to compliment all the things I learned from my
medical doctors with
things that I learned from holistic practitioners and so
even before my lam diagnosis, you know I sought out that sort of
Treatment and I keep searching and I keep dying you know I find somebody I got a guy now
I've been with him for a long time
And you know he helps me get through the winners without getting sick if something's going around school
Everybody's out put me. I'm there the only reason
I stay out is to go to Boston to a lam clinic
But I'm not sick so and I know if you get sick you get a respiratory infection
That's gonna. You know maybe you don't bounce back so much so I just think that doing things
you find useful and
You know not just supplements, but you know there's lots of things energy work
And you know there's there's lots of things out there
And you'll find your niche and you know that this makes you feel healthier
it makes your energy feel right and so then maybe you can fight off the things that are going around I could affect and
Damage the stuff that's going on I know this morning that some of the parents mentioned that when they change some diet
Or they did yoga or certain things with their children that they noticed changes
So I think we all can you know seek out those things not to discount any?
Other method but bring them together and see what works for you
So what what did was be Madeleine what um?
Well homeopathy works good for me. You know few little drops under my tongue who stressed now. What is it?
I
Do that I do you highlight. Go. I like secrets awesome. Yeah, Tommy asked me works out for me
Acupuncture has you know you you'll find that you do energy work. I teach my kids out of thump
You know you want a thump with me what we do some stress push it away
There's there's so many things out there, and you'll know what's right for you. You're like. Yeah, this is this fits me
Did you also have something yeah?
Yeah, so along that vein I think it's I do think it's essential that we really looked at non-drug treatments
For these things and that we consider how to incorporate them into our lives in a way that makes sense for us for me
yoga is a huge part of my personal treatment regimen on Yoga breathing to preserve my lung function I
Do a ton of deep breathing exercises of whether it's pulmonary rehab?
or is actually yoga either way because of course one of the problems that happens if you don't open up your lungs the the
collapsing of the lung is going to continue, so
and you're going to lose on function in that respect even if you don't lose it because of the cysts because so I just
Think that in terms of like keeping it from this holistic standpoint for me personally
Yoga has been huge to the point that even the Ps the operator at nih
Has made comments to me over the course of time about the amazing?
lung capacity that I have given the fact that I have Lam and
He's always amazed when I talk to about yoga is the way it's affected my lung capacity
So it's not even like a professional has made that connection as well
so I mean it's just something that I would really encourage everybody to be thinking about because there's
Even aside from that there's also the relaxation benefits and the mental and emotional health benefits that it brings
It's just something that we all should be considering
More strenuously incorporating into our regimens as my two cents
so only briefly state that I tried Depo-Provera very shortly for about a year at the very beginning and
stop that, and thankfully Nobody's talking about reviewing that but one thing that I am I do do and
I'm hoping we can come up with some more
Research information as to why it works. I spoke during my presentation about fatigue
What I use is long-acting
Bronchodilators, and it's a 12-hour one
I've tried a couple different ones
and
it's the only thing that's been able to get me through the day when my kids were young I would literally have to take a
Nap in the afternoon and my pulmonologist suggested, I thought why do I need a bronchodilator? And it was it was?
Described one time that
Our lungs have a hard time functioning the process of expelling air and inhaling etcetera, and I thought well. I'll try it
totally changed My Life I
Can tell if I've forgotten to take it in the morning by about one o'clock in the afternoon. I'm exhausted
I can feel the difference and I did participate in one study where we had to be off of it for a number of days
By the time I could finally take again. I was back to taking naps again in the afternoon
I don't know why it works. I hope we find a reason because it's something that I've heard a lot about sure
Nothing else that we haven't discussed yet that anyone wants to bring up
Yes
Every month I get a massage for my lung if medical trust me
medical massage
She she tries. She does lymphatic massage also to try to get the extra stuff out of the lymph nodes
But it is just she goes in between the ribs and gets those muscles, and it is the best feeling whatsoever
I mean, I fall asleep. It's so relaxing so I think everyone shouldn't try that
Does anyone else have experiences with things that they have tried that did not work?
Or where the kind of your personal benefit risk Led you to discontinuing a product I?
know a lot of
people had answered the response that they
Had found a product that stopped working for them. I'll take comment here, then we'll go to the web
Jean Daily I have sporadic Liam
I've
found that the long-acting
Bronchodilators has been helpful like Mary's suggested, but I found some pulmonologists are very
Hesitant to Prescribe the Bronchodilator without the Steroid attached and
The combination Steroid and long-acting bronchodilator has caused me problems
And I prefer the long-acting bronchodilator
We have a comment from Nikki she felt an adult with t. Sen lamb
She says that she tried doxycycline and that it did not work, and it also gave her esophagitis
Great. Thank you. Nikki
All right, then maybe we can now change gears to our
Question we talked a lot about what's been available in what you've tried so short of a cure
What specific things would you look for in an ideal treatment for you or the person that you care about?
So what would be meaningful to you?
as that next option
yep, I
Would love a Poc that runs continuous up to four liters that weighs less than 10 pounds
with a five-hour battery life
I
Don't know that we have anyone from the center for devices here
But I'm sure we can pass that along they might be on the web
Sue Sherman I'm the executive director of the Lam foundation
And I do not have lamb however interact quite frequently with the lamb community, and we recently did a survey to which
250 women with lamb responded and the question was
What affects you on a day to day basis most of these questions
We've been talking about today, and this is in preparation for a conference in the fall called patient benefit
And our goal is to
Address questions and come up with solutions that will help patients with things about that need to be solved in five years or less
So we have a long term goal of treatments and cures, but that takes a long time. How do we make life better now, and
the highlights of that are
some kind of Oxygen
Therapy and supplements that works and keeps you functional other answers were
anxiety and helped me with anxiety and depression
Helped me ask the questions. Help me communicate with my providers in a way that they don't think I'm crazy
we had a lot of
Help me remember, what our other topics were?
Fatigue with this crushing sense of fatigue that is different than not being able to breathe
help
right using devices to measure
Actually, what's happening with your disease whether it's home-based barama tree or the impact of nutrition or using fitbit types of devices?
To contribute to again this communication with your doctors so that you can let them know what's really going on with you
the impact of exercise
How much is enough how much is too much? What's good for my lungs? What's bad for my lungs?
The question is what can I be doing?
To stay healthy while I'm fighting this disease
How much of what you've heard up here and then I think that the fundamental question of which version of lamb do I have?
How is it going to progress am I going to have the kyle version am I going to have a multiple lung collapsed version?
How long will I be able to go without oxygen and what is my future look like?
So this is what you get from a well-educated
Highly engaged group of patients who want to contribute to an improved quality of life?
So I know on our panel we heard
Some different responses to this question we heard about the prevention of further decline to Avoid
Needing a lung transplant
We heard about I think that might have been booth of your time nicole and onsies comments
And then we also heard about wanting something that'll actually improve function
From Madeline, so I'm just curious from our audience. You know you personally with your own disease
Your own progression. What is it that you would look for and hope for?
On top of whatever it is that you have that has worked and hasn't worked
This is Mary again. Sporadically some something that I think needs to be addressed for me most obviously I want to cure
Without that I'd like the progression of the disease to be slowed through treatment, but but the other thing that I think is extremely
important is
dealing with how do we
confront
Pneumothorax is implored Eases
This is a huge problem in the group of us that have
recurring pneumothorax is and if you're a lam patient if you've had one you're going to have another one more than likely and
How do we address that how do we get our very well-meaning pulmonologists?
And/or Thoracic surgeons to understand that our loans are different. They do not react as other people's loans
They do not stay up with a simple little bit of spray here, and there we're dz's we need
aggressive treatment
Some don't want aggressive treatment that in
But we need to be involved in that treatment
And I've been a victim of aware what he thinks is a well-meaning thoracic surgeon who went in and did something?
but in the end
I'm probably going to have to have another Pleura Dz's because because he didn't do it as aggressively as he should have but he didn't
Ask me. He didn't even speak to the researcher that I asked him to speak to he made that decision on his own
So one of the things that we need to do to communities to help each other to learn how to be an advocate
which I am and it still happened to me and
to get the medical community to listen to us and
Start our clinicians that know lamb and our researchers that know lamb
Very important. Thank you. Maybe another way to think about this is also what?
activities were things that you
Maybe it's not at the falla measure of lung function or some other symptom
But what in your daily life would you like to be able to do again or continue doing that's important to you
that if you had a new treatment or therapy
That you that would be important like I said to either maintain or maybe even to get back
improve a little
let us know what what is important to you as
someone living with this yep, this is an McKenna, and I am the sister of
atSC lamb patient, and I think a treatment that
Completely stops the progression of the disease not something that has to be taken every day something that could
You know maybe not cure it
Maybe not make everybody better
But could come in and just stop the stop the disease something that you know sir alamos is wonderful
But after a period of time some people are finding like Madeline said that it's not working as well anymore
So something that we know will continue for the rest of her life
Yes
With love and right here the orange and then we'll go
So it's a long again
of course for me to be best to find a treatment for
Lam because okay 40s see I've had the kidney transplant I have
Let's say quite a lot of things things
Done, and I'm afraid of the future because mostly because I'm lam and not because of tse
So yeah to be I have tried for other treatments
back flowers
if you note, okay are quite a day to help you sleep or
being less dressed but of course that doesn't do miracles is a way, so
Again, I would love to find a treatment for Lam or at least know
What I can do what I cannot do and if the fatigue that I have now is it the lam?
Restarting or something else
So for quite comfortable drop out as well
Thank you
Jeanne Daily I was Braddock liam. I guess I wonder if there's any specific
efforts out there to develop
A
treatment that specifically targets the liam cells based on their
Let's say surface characteristics or something along that line. Is there a way that something that could be inhaled
Would attack the Lam cells
but not the
Normal lung cells, and I realized wrap up you know has effects on Liam cells, but it also affects
Other kinds of cells in the lungs, but is there something else about the lam cells that could be specifically targeted?
in a therapy
And could you explain why it is that you would want something to either not target the non lam cells?
What was what is the Earth? Do you have a specific experience?
with no, I don't think it's just you know wrap ahead side because it affects the immune system and
I'm just wondering if there's some characteristic of the lam cells like that surface antigen that they were describing. You know
Could you attach a therapy to?
Something that recognized HM
B45 whatever the number was and
Target something that fact right something that might help produce the side-effect profile
I'd like to see some treatments that actually enable people to revisit the idea of being able to have a family
and
to have a successful
Pregnancy and one that doesn't result in harm to the mother or the child
You know when I was diagnosed with Lam it
I was already sort of at the cusp of being a little too. Old to have another child anyway, but it literally
cut off any possibility of my being able to consider another pregnancy because I
Had a child already that needed my care and I could not take the risk of the house
You know my health going down going the future?
and
Besides that I couldn't even consider going off of sirolimus at that point because it was the only thing that was keeping me alive
but then for so many others like Sarah like
many land patients
And then other people in this room that have children understand what that means?
the ability to have children is
Fundamental to us as humans and if you want to have the child in you that's being cut off from you it there's no
There's nothing more profound in my opinion that's going to affect your life than that
So I'd love to see some treatments that enable that for the land community and the other communities
And to piggyback on that is because many people ask why my child is not going to adopt?
or have a surrogate and all that kind of stuff, and you really don't know what your life expectancy is going to be and
How much shortened it can be so is it you know?
I think that is something that really has you know people very worried
And if you listen to everybody here you understand that so if you're considering bringing a child and having a family
That's you know of tantamount importance
Hi, my name is trish, and my son David has Tuber sclerosis
He doesn't take any drugs for his for his condition and disease right now, and we're very blessed with that
But sitting here and listening throughout the day I've heard sort of a repeated theme
That I thought the FDa or though
Industry might address is these drugs are approved the gabapentin
the
Mtor inhibitors the Lam drugs, but the repeated theme is that people take them and they have great results?
but not forever
There is breakthrough seizures. I start regressing again
my lung function starts decreasing again after years of being stable
so
when you have these drugs, and they're approved is there any
ongoing research as to
what may cause their efficacy to fail or go down or
Is it once they're approved or on the market and it's up to you in your medical provider?
so
We're not posing questions
But but but when you're making, but when you're looking at decisions about treatment
You know that's it. That's a thought when you're thinking should I start taking this drug? Well? Yeah a lot of people have great
results with it, but then five years down the road they start having seizures again and
Do I want to risk?
The side-effects for maybe five years of a year three years of stability or maybe I'll get 20
these are considerations that
Healthcare providers and people affected with the disease will always think of sure and so I know
You know the information that you know fDA's decision is based off of this
Short term by comparison to you know a life-long of using these
Products, chronically and so you know fDA's decision is kind of at the time when it's ring benefits and risk you know at you know
The based off of the duration studied for clinical trials, and I think when we have diseases where there are unmet need
There's a desire to you know not have
long Outcome Clinical trials
You know in order to approve drugs
Generally, I would say that there's a continued monitoring mostly on the safety side, but not really
on efficacy, so unless there's
Additional research into the healthcare databases things like that that are observational in nature so following patients that are on the product
But that's there's no real kind of regulatory
purpose, I don't think
For doing that that would just be something that could be done
by other stakeholders
You know potentially payers would be interested in that from a cost-benefit perspective
And so I think that there is probably some
There are some databases that could be mined for that type of information, and if it's important to the you know treatment decisions potentially
Academia or even
Patient organizations could take that task on
But I don't know that that's something that you know falls cleanly within FDA is kind of the regulatory decision
Okay
Well inches Shannon and just in relation to that
I think that's what emphasizes importance of trying to find some new treatments and new tracks because we all know
We become immune to drugs as you only take it for so long when you start taking drugs at such a young age
Or you start in your 20s, and you're getting in your 30s and 40s and 50s
Your bodies will naturally either sometimes think I'm accustomed to the drugs and are you maxed out you can't have any more of it?
so I think that just sad just speaks to the importance of
Continuing that research and trying to find the new drugs that we have more options
for our adults and our children that got only lamb and PSP I
Think the point of additional drugs, but you know I think the point of duration you know of effectiveness
Is there's an important one as well?
right, so
Any final thoughts. I think we're at about time, but yes, please
We were discussing this morning about epilepsy
and I
Have a son I'm sorry. My name is Carla. I'm from italy. I have this 19 year old son
Who no longer has seizures, but initially we had many seizures
We try to figure batra and one of those people that unfortunately it didn't work with
it was available in italy, and it was like first my first-line treatment for
infantile spasms
I can remember that when my son was very young we were already told
that in the future we could
Encounter all the problems related to Talent. I think that that should really be
taken into consideration
With the neurologists when treating children with Trc
Because I think it's really important to have any kind of therapies that can go hand-in-hand with
it's not related to epilepsy with you know with the
Behavioral issues as the child gets older you have drugs that are available you have risperidone
perhaps for his adhd or you know for hyperactivity
for mental health issues
I think we're taking a bit of serotonin which takes
took as a hell of a long time to kick in because when when things happen they really happen and they happen bad and
To get them stabilized again. It always takes such a long time
And you also have the behavioral
therapies which are available and they could should work side by side, and I think that that
Isn't working properly because as far as I'm concerned
When I've been asking for five years
To have something like that to have that kind of assistance for my son um I don't think I'm the only one there
it really is unacceptable because you're
debilitating the carers at that point, so
Yeah, I think we should be looking into
As we were saying with Lam complementary therapies which are already available, especially when we have really difficult problems aggression
and
Mental health problems with TSI related to tse. And sorry the tongue issues
I'm sorry if that's a little bit garbled, but that's the way I speak very
Very important as you know some of even though luckily it sounds like seizures
Are no longer as large of a concern there's?
You know an array of other?
burdens that carry into adulthood
And whether that's being able to intervene in childhood, but also continue to treat into adulthood
Is very important relevant?
I
Think it slightly neglected across across the board and I'd really like that
to be taken into consideration
As well for a lot of the people and parents as we get older and I know there was another family here who are saying?
That they have really difficult issues
I think that it can help, but if you're waiting years and years and years that you get to a point when
you know you really don't you've got to the end of the road and
it's about it's about what's going to happen to them after we've gone as well, so and
There are things out there that can help so let's get that working together and networking
Thank you
That I think we will conclude our
moderated discussion before I
Thank you
it up perfectly so
Before we cry in the mic over
For our summary comments. I just want to thank all of you for your participation today
All are our panelists and panel - you are free touch you don't have to hang out on stage any longer
But I would like to thank all of our panelists
But I want to also thank all of you that. We're in the room participated in audience discussion and we're online
Participating as well. We couldn't have done this without all of your input, so give yourselves a round of applause I
just want to
Say you know I've worked in
patient engagement
In the regulatory process and have continued to do so and so from my perspective
I know that the input that you've heard that provided today is going to have an impact
I think you will hear about that from FDa shortly
But I also want to just thank you all and thank the ts alliance and Lam's foundation for letting me be part of your
extended Community
today and Also Beyond
So it's been an honor a pleasure to be your moderator today
and with that I will exit stage and
introduce our summary speaker from FDA
Dr.. Jonathan Goldsmith is the associate director for rare diseases at Cedars office of new drugs?
For those of you that we're here this morning
I'll let you know that he earned his medical degree from NyU and received his post-grad training in internal medicine at Vanderbilt and
Completed specialty training in Hematology at unC
he has had an extensive career in Academia as well as
as a tenured professor as well as in regulated industry where he's focused in Clinical drug development and
Has also worked with rare disease foundations so join me in welcoming Dr.. Goldsmith?
Right well, thanks again to all of you. I won't say the same thing. I said this morning although
I may go over a little bit just the important things I'll say twice
You know that repetition your teacher right? So that's good
so I want to just add my thanks on behalf of the FDA to the
organizers patients and families members of regulated industry academia government for including the FDA in your
Patient focused drug development meeting I think to make this a real success that has to include all the different pieces of the puzzle
And I think we're part of it
And you're part of it and to get us in the same room and to talk to each other
I think is always a very productive process I
I'm not going to do all this
Okay
Listening this afternoon. This is obviously quite different from what I heard this morning although
There's some similarities of course and it struck me
Your your insight and expertise to divide this into two sessions because it's kind of a bimodal
system, I mean as a whole set of disease issues and presents and children perhaps young adults and then many adult this is quite a
different
disorder it really has lots of different features and and you have challenges as a
Nonprofit
To address all of these for all of your constituents, but it was a challenge well worth taking on
So from the meeting today
We heard about your commitments and caring for your families
Your courage and Determination
and it's very heartwarming I can tell you I'm I'm
Very flattered to have been allowed to be in the room and to hear you speak
If you hear about what you've done, or what you've accomplished and what you will accomplish
We also heard about the heavy burden of the disease for you and your families
about the diagnostic journey
The delays and I would say I guess I call them medical insults
When things are not done correctly I don't know the right time I guess malpractice is the wrong time. I can't say that
But it's really it's very difficult for you
I know that you run into stone walls, and it's and people should be thinking when they talk to you
We heard about your respiratory
Complications with your central feature because of wam being a big component of the annals of the adults who are affected?
And and what the different approaches were that were taken
Some was it sounds like with better information than other
than others
But it sounds like your network is probably going to be helpful to you along the line to make sure
That the best decisions are made and you get the best advice from the best experts like you haven't
Well authority sitting here in the room and dr.. McCormick, and they could call him they could use the phone
Just tell him call this man, and I think he should
You know I think you should think about it. You know if you talk to other people as you meet who a disease affected
We heard about seizures that trickle on into adulthood and are just as troublesome as they were in childhood
we heard about
mobility issues challenges with daily living fatigue
Exhaustion I'm not sure quite what the right term is, but I heard a lot of those years and they are there clearly
Probably pretty easily demonstrable if you were asked to undertake some
Some functional exercise like the six-minute walk test at FDA's fond abusing
in the terms of assessing function
Because your lung capacity would be less and your exercise capacity would be less so is therapies come along
Maybe that's a an end point that we might look for in a clinical trial
We heard about the challenges with the activities of Daily of Daily living
The things that that people who are not disease affected take for granted
Like going to the store is a big deal for you takes planning, and it takes some energy
And you may not be able to do something else. You may be doing a trade-off or
Something later in the day or later in the week, you just give it up
That's sort of loud and clear
fatigue and weakness I
Heard about the complications and benefits of a surgery and invasive procedures and there clearly are both
It's not easy to do surgery when there are multiple lesions that the surgeon has to deal with and then
What's the best approach to those?
We heard about a managing complicated medical care at home
We heard about oxygen delivery and some small advances in the way that oxygen is delivered
And maybe I assume this is the best way for you to get oxygen from an oxygen tank. I'm not pulmonology
I don't know that, but I know they're
reprocesses in there, and they're
bigger machines that can be plugged into the wall at home that can extract
But I don't feel adequate for you or for your europe problems
we heard about prolonged hospitalizations weeks and months and
often
unsatisfactory at time of Discharge
It's like you were in the hospital for two months
And you're just as bad off as you went in the door the chief complaint was not addressed
It's unfortunate we knew we need to do better
We heard about the family impacts of the disorder and we heard about financial impacts
How it how it?
Robbed you of a lot of things you want to do or if we also heard about young person a young woman with lamb
Kind of turned her life around she's going to do it all now
Unplug and go up, and do what you want to do
These are tough decisions we also heard about
The losses that you have of a normal or near-normal life that
Episodically you may lose or on a permanent basis you may lose
And we heard about things that affect
Well at least
The way I think about about climbing
lifting
activities of daily living work living on the second floor the townhouse it's not practical I
Gave up the second floor a long time ago actually
It's not a bad idea
From technological benefits I heard about Oxygen delivery of liquid oxygen
Giving you some more independence. I didn't hear other things that was in that area
so
We heard about your views on new treatments
And those under development
regarding possible drug treatment effects and to prevent further
Decline a lot of times when we look at or think about drug development. We think about something
that's curative, but we hear a lot of
from a lot of patience and a lot of different disease categories about
Slowing the rate of decline and I heard that message again here today that that was
Something that would be acceptable to you in terms of the therapeutic
We heard about the desire for new treatments
with better risk benefit Profiles maybe longer effectiveness
periods
And also the consideration of complementary approaches
Complementary medicine as a discipline and so on and things that you can do in a general way to improve your health
I wanted to just repeat something I said this morning. We're talking about getting drugs to market faster
I just wanted to say again what I said this morning, because I think it's worth repeating
if you want to if you want to do a trial design in rare diseases you have a limited number of
Patients who are going to be able to be eligible for that particular study?
and
You have to make the best use of those studies if that human capital has to be used correctly and one of the problems that
Happens in drug development. Is that is that there's not a well-constructed study to evaluate
That particular agent
And then people have the opinion that it might work when what in fact it may or may not work
And then makes it impossible to actually do a trial that would lead to licensure
So if you randomized from the first patient you have people who are not treatment or more standard of care arm if you will
And keep that as short as possible that part of the study that will actually get your drug to market sooner
In terms of having controls for a study
If you as you or as your organization has been you try to develop registries to have natural history studies
and if these are well constructed
prospective and Gather information that drove down two important points that have to do with how
patients feel function and survive it may be very useful as an external control for a particular study and
third
Biomarkers are important. You know. What is the biomarker this morning? We have the eeg with a biome or
you know and there has to be a way that that the
Biomarker and and the disease progress have to be correlated the rest to be a validation does it really you know
What's it really marking? What does it really predict and the way the biomarker is done like let's say
It's a blood test for some protein
Well you have to be able to do that assay over and over again
and it has to be a standardized way to do the acid a lot of these are developed an academic setting and
They don't transfer to another why we're doing that's one of the places where that gets in trouble
But this should all be addressed early on drug development, not when you're trying to get into a face
We're studying and you're at the end
And then you're kind of in a difficult place
There's also some some work that we're going to do to help growth developers in the based on the 21st century cures acts
I'm sure you've all read that, right?
Tiny Little document
anyway, so
We're going to develop and put under promulgate guidances and rare disease drug development
We've already issued one on rare diseases or common issues in Drug Development in August 2015
We hope to finalize it in the near future
We're also going to issue Guidance is according to the 21st century cures which means we will do it as long for
Formalization of patient preference studies and submission of draft guidances which some which some stay cold some stakeholder organizations?
Have been doing already
And the last thing I guess that I wanted to say something about about whether what happens post marketing
And you know fdA Fda had kind of limited post marketing authorities till about?
2007
But now has pretty substantial post marketing authorities, and you know we asked firms
To do safety follow-up. That's required by law, but we also may ask them to do additional studies to find out
Does the drug work perhaps?
In the population that was stayed in the clinical trial what happens when it's used in a bigger population
That's what happens with all drugs. Is that there's a clinical trial developed just
Inclusion/exClusion Criteria, it's highly selected some people might say it's contrived
And that that's the grooved afeni fitted now when you pull it out
and you give it to the general public it may not be quite the same, so
Although FDa doesn't doesn't really focus in on exactly what you were saying
These may be issues that you'll want to take up when you talk to people at FDA over the years that you want to have
Some kind of assurance that that the drug works for a long time
it's kind of a different concept, but when we like to hear more about and think about and
Are the ways to predict who who's actually not benefit for the longest you know what can be done?
Scientifically, and maybe the drug developer will look into that. We you know we don't actually develop drugs. You know at FDA
We just regulate them just just to make that clear
Okay, so again. I want to thank you very much for including FDa today
You know it's a pleasure to talk with some of you I
Really enjoyed hearing your stories. I'm a physician at heart, so I have
Absolute Empathy with you and it was really just a great day. Thank you
Thank you, Dr.
Goldsmith and thank you for all of you at FDA who spent the day with us and those of you those of those who were
Here in the morning
And I know there are many engaged on the web who have been watching it would be it would be
pointless without your presence your participation and really taking all of this in
And thank you, especially to those who participated because it would be absolutely nothing
Without you and what you've brought to the conversation today
This isn't the end so it's the end of today's meeting
And I think you all are probably comfortable ready to go do something else
But it but it's far from the end of the process, so we will have on the ts Alliance org Slash
PFd D
we will be posting a link to the recording of this webinar, and we will have a link to a
survey Monkey
Location where you can enter comments?
So those of you who are in the room and didn't get to see everything you wanted those of you who are online?
Who would like to contribute comments that we will read and incorporate into the voice of the patient report?
And you know those who are viewing this as a recording who didn't see it feel free to
To go to ts
Alliance org Slash Pfd D and find that link and enter your comments that will be open until july 24th at 9 a.m.
and then we're going to start reading the comments and and putting them to paper and that will result in a written report to the
FdA
the voice of the patient report that will document and summarize all of this together with
With the survey results that that we've collected
As a reminder before we close, please leave your clickers
the
Remote control things leave them in the chair
Drop them off at the table. Whatever your lanyards and badges. We'd be happy to
Recycle if you leave them at the table
All of that saves a little bit of money
Especially the clickers those are expensive if we have to pay to replace them, and that's money that wouldn't go to research
so please leave your
clickers
so
Finally I want to thank all of our speakers our panelists the individuals with PSC and lam and their caregivers for joining us today
We're most grateful to the representatives from FDA for being here to represent us some industry who joined
To everyone who absorbed and shared their perspectives
This is a crucial step for drug development and tSC and lam and we look forward to continuing to work together
To improve the quality of life of all of those living with tsE, and lam. Thank you all have a great
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