CureDuchenne and Prosensa to Present Collaboration at Partnering for Cures Conference

Newport Beach, Calif., NOVEMBER 12, 2014 – CureDuchenne and Prosensa Holding N.V. (NASDAQ: RNA) have been selected to present at the 6^th annual Partnering for Cures, November 16-18 in New York City. Convened by FasterCures, the Washington, D.C.-based center of the Milken Institute, the conference will bring together nearly 1,000 medical research leaders, investors and decision-makers to forge the collaborations needed to speed and improve outcomes-driven R&D. CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, and Prosensa, the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, will present “Accelerating Access to Treatments of Duchenne Muscular Dystrophy” on November 17. The presentation will focus on CureDuchenne and Prosensa’s $7 million (€5M) collaboration.  

Partnering for Cures is designed to facilitate informed investments and cultivate relationships, adapting the outcomes-oriented approach of investor conferences, and building on the networking opportunities at industry partnering meetings. In addition to innovator presentations, it also features panels that spotlight solutions to long-standing challenges in medical research.

“We are proud to participate in Partnering for Cures and share our collaboration with Prosensa,” said Debra Miller, founder and CEO of CureDuchenne. “Our collaboration with Prosensa is an example of our venture philanthropy to help speed up the drug development process. We are committed to help accelerate access to drug candidates to boys with Duchenne.” 

CureDuchenne and Prosensa are one of 30 innovators presenting their cross-sector research collaboration to potential partners and funders at the conference.  Selected through a competitive proposal process, each partnership is aimed at reducing the time and cost of getting new medical solutions from discovery to patients.

“These collaborations address some of the thorniest issues in medical research using models that can be scaled and translated across diseases,” said FasterCures’ Executive Director Margaret Anderson. “From re-imagining clinical trial infrastructure to improving and expanding data sharing, to creating the tools and resources needed to translate basic science into cures, they are accelerating the path from lab to market for novel – and needed – therapies.”

Duchenne is a progressive muscle-wasting disease that impacts one in every 3,500 boys. Boys are usually diagnosed at age 5, are in a wheelchair by 12 and most don’t survive their mid-20s. Currently there is no cure for Duchenne.

For more information and to register for the conference, go to www.partneringforcures.org

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.

About Prosensa Holding N.V.

Prosensa (NASDAQ: RNA) is a biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Its primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy (DMD), myotonic dystrophy and Huntington’s disease.

Prosensa’s current portfolio includes six compounds for the treatment of DMD, all of which have received orphan drug status in the United States and the European Union. The compounds use an innovative technique called exon-skipping to provide a personalized medicine approach to treat different populations of DMD patients. www.prosensa.com

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