[Congressional Record Volume 162, Number 66 (Thursday, April 28, 2016)]
[Senate]
[Pages S2534-S2535]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




                      DUCHENNE MUSCULAR DYSTROPHY

  Mr. BARRASSO. Mr. President, I rise today as a Senator and, as the 
Presiding Officer is, a doctor. I want to talk about a disease called 
Duchenne muscular dystrophy. Earlier today Senator Rubio was on the 
floor talking about the disease, and I know earlier today Senator 
Wicker was on the floor talking about the disease. It is a topic that 
is, as an orthopedic surgeon, very personal to me.
  I was introduced to Duchenne more than 30 years ago and, as an 
orthopedic surgery resident, worked at a muscle disease clinic with 
young people with muscle disease. One of those muscle diseases is 
called Duchenne. It is a disease that affects young boys. I met 
patients and I met their families in the fight against this disease. 
The experience has left a lasting lifelong impression on me, and it is 
something I continue to work with today.
  I think the reasons we have gone into medicine are to help people and 
to make a contribution. One of the reasons I chose orthopedic surgery 
was that I really enjoyed seeing the relief--the care that I gave could 
help people, causing relief of their symptoms, relief of their pain, 
relief of problems they were living with from day to day. It is 
extremely rewarding to be able to work with a patient and tell that 
patient the surgery you performed was successful, and they are going to 
get better. They are going to get back to normal.
  As a doctor, I was able to see patients go on to graduate from 
college, get married, have children of their own. When I was overseas 
visiting our troops, I met a young man, a commander--a pretty big guy--
and he told me I had taken care of his broken leg. I looked at him and 
didn't really recognize him. I said: When was that? And he said: I was 
only 8 at the time.
  We take care of patients and, as we do, we see people through their 
lives, and it is encouraging to see them go on and strive and get 
stronger and bigger and more productive. But for patients with Duchenne 
muscular dystrophy, that kind of treatment doesn't exist. It doesn't 
exist today with all the breakthroughs and research.
  When I saw patients in the muscle disease clinic who suffered from 
this condition, I knew the day that I saw them was going to be their 
best day from there going forward. Many of them had brothers. It is a 
disease that affects young men. It is a disease that may be coming in 
their family to children who had not yet been born. In some families 
there were several brothers in the line who had the disease. As one was 
diagnosed, then another younger brother was diagnosed a couple of years 
later with the same disease because this does tend to run in families.
  As a doctor, one wants to see somebody get better and stronger every 
day. Parents want to see their own child going from crawling to walking 
to running, getting stronger and bigger every day, but patients and 
families who live with this disease every day know too well the 
unrelenting force of Duchenne muscular dystrophy. What it does is cause 
degeneration of muscles and weakness.
  The vast majority of people with this disease are boys, and they are 
usually diagnosed between the ages of 3 and 5. Typically, parents start 
to notice their son isn't meeting all of the developmental milestones 
they might expect. He might be a late walker, or he may appear less 
coordinated than other children his age. Most parents aren't worried; 
they are just cautious. They may mention it to the pediatrician, and 
the doctor may run a test or two. Once the diagnosis of Duchenne 
muscular dystrophy is made, patients pretty quickly and parents, 
specifically, very quickly find out that their son doesn't just have a 
developmental delay; they learn their son is typically going to lose 
the ability to walk by the time he is a teenager, graduate to a 
wheelchair, which then can make that young man prone to conditions like 
scoliosis, a curvature of the spine often requiring surgery to correct 
it. As the muscles continue to deteriorate--as they always do with 
Duchenne--that young man will lose lung function, which puts him at a 
higher risk of infection, pneumonia. Eventually, he will have to use a 
machine to breathe, to clear his lungs. The muscle deterioration 
doesn't just occur to the skeletal muscles--the muscles of the arms and 
legs--but also can occur to the heart, which is itself a muscle.
  When a young man with Duchenne muscular dystrophy catches a cold, it 
can be life threatening. Even when the patients get the best medical 
care--and so many of them do get the best medical care--they usually 
lose their fight against Duchenne muscular dystrophy by the time they 
are in their 20s. That is the devastating reality of this disease, and 
we cannot allow it to continue.
  Because of my experience with these patients, I have been working for 
years to actually help raise money for awareness for muscle disease and 
treatment for the disease. I served as a local host in Wyoming for the 
Muscular Dystrophy Association's annual Labor Day telethon.
  Every year, I was amazed at the dedication and the generosity of 
people around the country who would call in pledges to pledge centers 
at the 200 so-called ``love networks'' in Casper, WY. People would call 
in. We would always raise over $100,000. People were very committed to 
finding a cure for muscle disease and to sending young people with the 
disease to summer camp, where they found a level of freedom and 
friendship that they did not often find throughout the rest of the 
year. It was a great time for the young people with the disease. It 
gave their parents a rest as well.
  I think many of us in this body remember Jerry Lewis hosting the 
Jerry Lewis Labor Day Telethon, as it was called, for more than 40 
years. He would always end the telethon by signing a song. The song was 
``You'll Never Walk Alone.'' So I come to the floor today to make sure 
that these patients and these families know that today they are not 
alone. Congress is listening. We heard from Senator Rubio earlier today 
and we heard from Senator Wicker. Those families and those patients 
know how critically important it is, and we know how critically 
important it is that we find a cure for this rare disease known as 
Duchenne muscular dystrophy.
  In 2012, Congress passed the Food and Drug Administration Safety and 
Innovation Act. One of the key parts of this law gives the FDA more 
flexibility to approve treatments that have the potential to help 
people with rare diseases. It also allows the FDA to do followup 
studies to confirm the clinical benefits of the treatment.
  Well, we want to give people real hope. It is not good to give people 
false hope. We are interested in giving patients and giving families a 
fighting chance. I believe the FDA needs to use the tools that Congress 
has given them so patients can come across and get access to 
potentially lifesaving drugs. So a couple of weeks ago I signed a 
letter that was written by Senators Wicker and Klobuchar--a bipartisan 
letter. It called on the FDA to take full advantage of this accelerated 
approval authority.
  So we also asked the FDA to ensure that the prospective of patients 
is fully considered in this review process, when it comes down to the 
regulations. More than 20 Senators signed this letter because we know 
how important this issue is to patients as well as to their families.
  Last Friday the Wall Street Journal ran an editorial entitled: ``The 
FDA vs. Austin Leclaire.'' This article talked about a young man named 
Austin Leclaire, 17, who has Duchenne muscular dystrophy, and so does 
his younger brother Max. As we talked, I mentioned that this runs in 
families. Sometimes, there is the diagnosis of a son in a family in 
which there is a younger son who has not yet been diagnosed but likely 
will have the disease.
  Well, back in 2011, Max was able to get an experimental drug to treat 
his disease. Now, Austin was not eligible to get the same drug. 
Remember, Austin is the older brother. So today Max is 14 and he is 
still able to walk. He can

[[Page S2535]]

still play sports, and he can still dress himself.
  For most of us who have had healthy children, these are the things 
that people take for granted. So for a family where one of their sons 
has Duchenne, this kind of small victory can seem like a miracle. I 
can't even imagine how hard it must be when a mother has two or three 
children--two or three sons--with this disease, and especially when one 
of her children can get access to an experimental drug and the other 
cannot.
  The family looks at it. One son is being helped, and the other is not 
being helped. They can see the difference in their sons. So how would 
any of us here in the Senate react if we were in that same situation? 
How much heartbreak should one family have to bear? Those are the 
challenges for families who live with muscle disease every day.
  Well, the FDA, I believe, needs to work with patients like Austin and 
Max. We all know that this agency needs to make sure that treatments 
are safe and effective. That is not a question. We also know that 
people at the FDA are caring and careful professionals. The practice of 
medicine relies on hard science and on following data to understand and 
to treat illnesses.
  As a doctor, I know that the practice of medicine requires an equal 
measure of compassion. I think the FDA needs to take into account the 
unique needs of this patient population. We talk about double-blind 
studies, where you give one patient the real treatment and one patient 
something else, a sugar pill, something else that is not really the 
real treatment, the real medication.
  To really evaluate the impact of these medications, sometimes it 
involves doing muscle biopsies and putting people though painful tests. 
I think it is hard for a family living with a child with muscle disease 
to say: Well, we are going to participate in the experiment. We don't 
know. It is a 50-50 chance if our child is even going to get the real 
thing. But we still put them through all of these tests that can be 
painful, as they take muscle biopsies.
  I think it is unrealistic to ask a family to make that decision. I 
think we need to make sure that the FDA--and the FDA needs to make 
sure, in their compassion--doesn't lose sight of these kids. These 
young people really don't have a moment to lose in terms of potential 
treatments. I think the FDA needs to hear the calls of patients and to 
give these young people, living with a devastating disease, a chance to 
beat Duchenne muscular dystrophy.
  I yield the floor.
  The PRESIDING OFFICER. The Senator from Oregon.

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