Newport Beach, Calif., DECEMBER 10, 2014 – 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 those impacted with Duchenne.”
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.”
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.