|[September 20, 2013]
CureDuchenne Media Statement re: Phase III Clinical Trial Results for Drisapersen, GlaxoSmithKline/Prosensa Therapeutic's Treatment for Duchenne Muscular Dystrophy
NEWPORT BEACH, Calif. --(Business Wire)--
"While we are disappointed by the Phase III trial results for
drisapersen, we hold out hope that they will provide critical
information to the research community that can be applied in future
studies, including those by GlaxoSmithKline and Prosensa," said Debra
Miller, CEO and Founder, CureDuchenne. "We are fully confident in the
exon skipping technology as a viable platform to develop a treatment for
Duchenne, and Duchenne families should not give up hope."
In addition to its support of Glaxo and Prosensa, CureDuchenne is
working with multiple companies to develop a robust pipeline of
potential drugs to treat Duchenne. CureDuchenne also provided early
support to Sarepta, another company in trials for its exon skipping
drug, eteplirsen. Currently, there is no treatment for Duchenne, a
progressive muscle-wasting disease and the most common and lethal form
of muscular dystrophy. Duchene impacts one in every 3,500 boys; nearly
20,000 boys are living with the disease in the United States. Boys with
Duchenne are usually diagnosed before the age of 5 and are in a
wheelchair by age 12; most don't survive their mid-20s.
is a national nonprofit organization 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, the organization has raised more than $11 million
towards promising research aimed at treating and curing Duchenne. In
fact, 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. For more information on how to help raise awareness and funds
needed for research, visit www.cureduchenne.org.
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