CureDuchenne,
a nonprofit that raises awareness and funds research to find a cure for
Duchenne muscular dystrophy, will continue to watch the performance of BioMarin
Pharmaceutical Inc. (NASDAQ:BMRN) during its acquisition of Prosensa,
a biotechnology company CureDuchenne has supported at critical times.
Today, BioMarin is hosting its first Analyst & Investor Day since the
acquisition announcement November 24, and will be providing updates on
the company's pipeline and commercial programs.
CureDuchenne uses venture philanthropy – offering venture capital to
pharmaceutical companies – to help fund Duchenne research. CureDuchenne
was an early supporter of research into exon skipping, (a method used to
cause cells to “skip” over faulty sections of genetic code, leading to a
shorter but still functional protein), and has played a critical role in
helping to fund Prosensa’s exon skipping drug drisapersen. CureDuchenne
is invigorated by BioMarin’s commitment to continue the development of
such drugs.
“CureDuchenne looks forward to working with BioMarin, and we support
their efforts to further the development of treatment options for boys
with Duchenne,” said Debra Miller, Founder and CEO of CureDuchenne. “We
continue to use our venture philanthropy model to fund research to help
accelerate access to drugs and expedite the approval of innovative
therapies to treat this generation of Duchenne boys.”
CureDuchenne has supported Prosensa since the company’s inception and
funded Prosensa at critical times in the development of new drugs.
Drisapersen is currently under a rolling review as part of the New Drug
Application process. CureDuchenne recently funded other exon skipping
compounds that are under development.
“We are dedicated to funding research to find a cure for Duchenne,” said
Miller. “For the Duchenne community, the science can’t move fast enough.
With BioMarin’s experience in the orphan rare disease space we are
hopeful that there will soon be an approved treatment for this
devastating disease.”
For more information on CureDuchenne go to www.cureduchenne.org
or call 949-872-2552. Follow us on Facebook,
Twitter
and YouTube.
About CureDuchenne
CureDuchenne
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. 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.
Copyright Business Wire 2014