a nonprofit that raises awareness and funds research to find a cure for
Duchenne muscular dystrophy, will host a webinar
on accelerated access for drug approvals with the Food
and Drug Administration (FDA) on February 20, 2013 at 4 p.m. EST for
Duchenne parents and advocates. Dr.
Robert Temple, Deputy Center Director for Clinical Science, Center
for Drug Evaluation and Research, FDA, will present and discuss
expedited pathway programs, legislative initiatives and expanded access
to investigational drugs. As the most common and lethal form of muscular
dystrophy, Duchenne impacts 1 in 3,500 boys. There is currently no cure
CureDuchenne is hosting the webinar in collaboration with Parent
Project Muscular Dystrophy and the Muscular
Dystrophy Association to enable the Duchenne community and the FDA
to come together to further understand accelerated approval for
potential Duchenne treatments. Boys with Duchenne are usually diagnosed
before the age of 5, in a wheelchair by age 12 and most don’t survive
CureDuchenne has a sent a letter to the FDA on behalf of the Duchenne
community requesting attention to the risk tolerance that patients and
parents have for this fatal disease. CureDuchenne is the leading
organization funding research through venture philanthropy to find a
To register for the “Expedited Pathways and Expanded Access” webinar
with the FDA on Feb. 20 click here.
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is a national nonprofit organization located in Newport Beach, Calif.,
dedicated to finding a cure for Duchenne, the most common and most
lethal form of muscular dystrophy. As the leading genetic killer of
young boys, Duchenne affects more than 300,000 boys worldwide.
CureDuchenne has garnered international attention for its efforts to
raise funds and awareness for Duchenne through venture philanthropy.
With the help of CureDuchenne’s distinguished international panel of
Scientific Advisors, funds raised by CureDuchenne support the most
promising research aimed at treating and curing Duchenne. To date, seven
CureDuchenne research projects have made their way into human clinical
trials – a unique accomplishment as few health-related nonprofits have
been successful in being a catalyst for human clinical trials.