(the following is a letter I sent to the Township of Langley Firefighters)
Doug
is my 13 year old son, and he has Duchenne Muscular Dystrophy (DMD). You may
have met him at the Langley Firefighter’s Appreciation Banquet this year.
For
many years, the Langley Firefighters have joined with firefighters around the
world to raise funds to support families like ours. We appreciate it greatly.
Without the support of groups like yours, Muscular Dystrophy would be an
impossible battle to face alone.
Since
the time he was just 8 years old, Doug has been involved in a medical study for
a new drug aimed at dramatically slowing the progression of DMD. A typical DMD
boy will transition to full time wheelchair use by the age of 11 or 12, and has
a lifespan of 20-30 years. This new drug is looking to change this.
Doug
joined the study in the summer of 2011. For the next 3 years he made weekly
trips into Children’s Hospital for drug injections and testing. It was a
painful ordeal – each injection hurt, and the batteries of blood draws, MRIs,
EKGs, muscle biopsies, and other treatments were exhausting. But there was
promise. Where decline was expected, we found stability. Instead of moving
towards a wheelchair, Doug’s walking distance got longer and longer. His
balance improved, and at the age of 11, instead of riding in a wheelchair, he
was able to go on walks in excess of 2 miles long.
Then,
the drug changed ownership. During that period, the study was halted, and in
the 18 months it took to get restarted, we watched our son decline. We weren’t doing
long walks anymore – a couple of blocks was Doug’s limit. He began to need to
crawl up the stairs instead of walk. Our hearts broke.
A
couple of months before Doug’s 13th birthday, the study started up
again. Doug once again was on the medication. After a few months his decline slowed,
and after 6 months in, it seems to have halted. We have met with other boys who
have gained back some or even most of their lost time. We are hoping that as
time goes by the medication will help restore Doug’s lot time too.
The
FDA is deliberating approval of this game changing drug. It is called
Drisapersen. They aren’t sure what to do. The effects in the statistical data
are not matching up with what families like ours are seeing. About 20 families
like ours travelled to Washington DC to make our plea in person last month. The
drug must be working for so many people to have such dramatic evidence.
The FDA has a timeline
of Christmas Eve to decide on whether to allow this drug onto the market. This would
make it the first and only treatment for DMD. We need your
help to convince them that patients who have proven this drug works should
continue to have access – and to allow boys who couldn’t participate in the
clinical study access to this life saving drug. Without it, there are no other
options. This drug will not work for every DMD boy – but surely those who
respond to it should be allowed continued access!
Please sign this
change.org petition.
https://www.change.org/p/tonya-carlone-sos-sign-to-save-our-sons-give-our-duchenne-boys-their-christmas-miracle
This petition was started by Gavin’s mom. Gavin is a friend of Doug’s who was
in the same study at Children’s Hospital. His story is just like Doug’s.
Nearly
a year ago the FDA was dragging their feet on whether or not to even review
this drug. A petition started at the Whitehouse managed to raise over 100,000
signatures over the 30 days it was open. Shortly thereafter, the FDA accepted
Drisapersen for review. Your voice makes a difference.
Please
sign the petition and share it. I can’t think of a better Christmas gift for
Doug, Gavin, and all the other DMD boys.
Cam
Penner
(Doug’s
Dad)
