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New drugs are being developed to treat Duchenne Muscular Dystrophy — and many other rare diseases — but the treatments are stalled at the FDA. A new Kickstarter campaign is raising funds to complete a documentary about parents fighting to stop Duchenne Muscular Dystrophy and gain access to potentially life-saving drugs before the disease kills their children.
Oscar winning filmmakers Jedd and Todd Wider from Wider Film Projects are working on a new documentary called To the Edge of the Sky about parents who are fighting to save the lives of their sons who are dying from Duchenne Muscular Dystrophy.
Duchenne Muscular Dystrophy — one of nine types of muscular dystrophy — is a genetic disorder characterized by progressive muscle degeneration and weakness. It is caused by mutations in a gene called dystrophin (gene symbol: DMD), the largest gene found in nature. The dystrophin protein encoded by the gene is found primarily in skeletal and cardiac muscle, and acts as an anchor to a group of other proteins that work together to strengthen muscle fibers and protect them from injury as muscles contract and relax.
With genetic mutations in dystrophin, Duchenne Muscular Dystrophy or “DMD” occurs at a frequency of about 1 in 3,500 new-born boys. The symptoms of DMD present in early childhood, usually between ages 3 and 5. In most cases, children with DMD die before their mid/late 20s.
There are promising new drugs that offer the hope of possibly treating Duchenne. However, these drugs remain stalled at the FDA — in particular, eteplirsen (Sarepta Therapeutics), a Morpholino antisense oligomer (a molecule in a particular structural family that is used to modify gene expression) that results in a functional dystrophin protein . As a result, only a limited number of boys who are enrolled in specific clinical trials have access to them. One of the families in the documentary has two boys with DMD: one getting the experimental therapy is thirving, one can’t get the therapy because he isn’t in the trial. Their mother is scared that she could have the first child in history to survive DMD and the last child to die from it, living together in the same house.
Imagine if you were the parent of a child with DMD. Every day you knew you were going to outlive your child. Then, after years of watching your child struggle with a rapidly progressing deadly disease, a new drug offered you the hope of halting its progress. What would you do to obtain the drug for your child? What would you do to save your child’s life?
To the Edge of the Sky offers an unflinching portrait of four families going through just that, waging an extraordinary campaign to convince the FDA to allow the accelerated approval for a drug they believe is a potential cure for DMD before it kills their sons.
With incredible access to the families, legislators, and FDA, the documentary presents the day-to-day combat and strategizing required to get the government and public-at-large to pay attention to any disease, let alone one that affects just a few thousand kids yet creates tremendous devastation.
The filmmakers are raising funds on Kickstarter to accelerate their post-production schedule to complete the documentary. In addition, they hope a public forum like Kickstarter will allow people to see the struggles that these boys and their families face every single day. Moreover, they hope it will help convince the FDA to pay more attention to the special needs of people that are suffering from rare diseases.
Here at Highlight HEALTH, we think the documentary To the Edge of the Sky truly has the potential to change the world. In 1992, AIDS activists were successful at getting the FDA to allow for accelerated approval of HIV treatments by demanding change. Imagine what would happen if the FDA changed it’s rules on accelerated approval for DMD and other rare diseases.
The filmmaker’s goal is to raise $125,000, so if you think access to potentially life-saving drugs is important, go back the project right now. In doing so, you may very well save or extend the lives of many children with DMD or people with other rare diseases.
- Duchenne Muscular Dystrophy Statement. U.S. Food and Drug Administration. 2014 Oct 30.