(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.