Statement from CureDuchenne Regarding Prosensa IPO

Newport Beach, Calif. – CureDuchenne, a leader in raising awareness and funding research to find a cure for Duchenne muscular dystrophy, congratulates Prosensa Therapeutics BV on its initial public offering. CureDuchenne has been a longtime supporter of the company, providing early-stage funding 10 years ago for Prosensa’s antisense (exon skipping) research that led to the development of drisapersen, a promising novel drug for Duchenne. The U.S. Food and Drug Administration recently granted Breakthrough Therapy designation to drisapersen. Drisapersen has shown promising results in Phase II clinical trials released in early 2013 and CureDuchenne looks forward to seeing Phase III data later this year.

CureDuchenne was the first muscular dystrophy organization to recognize the potential of Prosensa’s exon skipping research and in 2004 committed $1.3 million to further that research. A few years later, venture capital firms validated CureDuchenne’s investment with several million dollars to continue their research. In 2009, GlaxoSmithKline committed up to $650 million to put four of Prosensa’s exon skipping compounds on the research and development path.

“CureDuchenne saw great potential in Prosensa’s leadership and research teams 10 years ago and is proud to have been an early supporter of the company,” said Debra Miller, CEO and Founder, CureDuchenne. “Our early-stage funding of Prosensa’s exon skipping research helped lay the critical groundwork for the development of drisapersen, which holds promise as one of the first effective treatments for Duchenne.”

Currently, there is no treatment for Duchenne, the most common and lethal form of muscular dystrophy, which impacts one in every 3,500 boys; nearly 24,000 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 www.cureduchenne.org.