TUCSON, Ariz., Aug. 15, 2011 /PRNewswire-USNewswire/ — More
than a decade of targeted Muscular Dystrophy Association-funded
research, made possible as a result of generous public support of
the MDA Labor Day Telethon and thousands of grass-roots special
events, has today culminated in MDA providing financial assistance
for the start of the first Phase 2 placebo-controlled, multiple
dose efficacy, safety, tolerability and pharmacokinetics clinical
trial of an exon-51 skipping drug, eteplirsen, as a potential
therapy for Duchenne muscular dystrophy (DMD).
The first three of 12 DMD boys participating in the AVI
BioPharma clinical trial at Nationwide Children’s Hospital in
Columbus, Ohio today received the first of 24 weekly doses of
eteplirsen or a placebo by intravenous infusion (i.v.). Four
more participants had muscle biopsies vital to measuring the
presence of the essential muscle protein dystrophin both before and
after treatments. The seven boys traveling in for the study
launch are from Calif., Ill., Fla., Wis., Va. and Mo.
“This is an important day for families fighting muscular
dystrophy,” said R. Rodney Howell, M.D., Chairman of the MDA Board
of Directors. “AVI BioPharma already completed a 19-patient
clinical trial in the United Kingdom confirming the potential of
eteplirsen to be a safe and effective disease-modifying drug for
DMD (The Lancet, July 25, 2011). Now, a team led by Dr. Jerry
Mendell is receiving funding from MDA to help initiate this
randomized, double-blind, placebo-controlled 12-patient trial
needed to further test safety, efficacy and optimal dosing.”
“Twenty-five years ago, MDA-funded investigators identified the
dystrophin gene that, when mutated (or defective) causes Duchenne
muscular dystrophy as well as the somewhat milder Becker muscular
dystrophy (BMD),” explained Mendell, Curran-Peters Chair of
Pediatric Research at Nationwide Children’s Hospital, and Professor
of Pediatrics and Neurology at
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