NORD hosted the 2016 Rare Impact Awards to bring together the rare disease community and to honor and celebrate outstanding achievements. Last night, Debra Miller, founder of CureDuchenne, was one of the esteemed honorees who was recognized for moving us forward in the fight against rare diseases. Congratulations Debra for your tireless effort to find a cure for Duchenne. Debra’s remarks are below.
Your child will be confined to a wheelchair by the time he is 10 and he will probably not live past the age of 18. And, there is no cure and no treatment. It’s best that you go home and love your child. Those are the words that changed our life when our son Hawken was diagnosed with Duchenne muscular dystrophy. I am proud to be among many others here tonight who heard similar words, but have chosen to fight to make that hopeless prognosis an ancient memory.
CureDuchenne has grown around problem solving…identifying what stands between us and a cure, and innovating to come to a different, and better outcome by applying new ideas, models and technology.
Using a venture philanthropy model, we allocate funds toward promising treatments and strategies to support the Duchenne community, including the following ways: We committed to get experimental drugs, shown to be effective in mice, into human clinical trials. We funded cTAP, a program to gather natural history studies and apply statistical analysis to help biotech and pharmaceutical companies design better clinical trials so the FDA understands the variation between Duchenne patients. We created CureDuchenne Cares, to take physical therapist and parent training on the road so that patients everywhere can have the best quality of life. We designed a comprehensive research portfolio to treat the whole disease and work toward a cocktail of drugs because many targets need to be addressed with multiple drugs.
I thank the CureDuchenne team; we have true experts on the science side who give us the best possibility of success. Thank you to the biotech and pharmaceutical companies that brave the world of rare diseases. I’ve seen firsthand the true compassion and commitment they have to the patients.
NORD, thank you for creating a forum for collective wisdom and experience. Time is not on our side as parents and patients, and we do not need to reinvent the wheel when can learn from each other. I accept this award on behalf of many, many families that are all contributing pieces to this puzzle called a cure for Duchenne and all rare diseases.