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CureDuchenne Media Statement re: Phase III Clinical Trial Results for Drisapersen, GlaxoSmithKline/Prosensa Therapeutic’s

  CureDuchenne Media Statement re: Phase III Clinical Trial Results for
  Drisapersen, GlaxoSmithKline/Prosensa Therapeutic’s Treatment for Duchenne
  Muscular Dystrophy

Background: GlaxoSmithKline (GSK) and Prosensa Therapeutics today announced
the results of Phase III clinical data for drisapersen, an exon-51 skipping
compound for the treatment of Duchenne muscular dystrophy. The data did not
meet its primary endpoint of a statistically significant improvement in the 6
Minute Walking Distance (6MWD) test compared to placebo. GSK has indicated it
would evaluate the outcome to help inform its next steps for drisapersen.
Prosensa said it remains committed to the drug development program and will
continue to work closely with GSK.

Business Wire

NEWPORT BEACH, Calif. -- September 20, 2013

“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. Duchenne 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.

About CureDuchenne

CureDuchenne 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


Karen Harley, 949-872-2552
Stephanie Blank, 646-805-2019
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