Drug approved by the FDA
for the treatment of Duchenne.
CureDuchenne was an early funder.
CureDuchenne funded research projects
have advanced to human clinical trials
Our lives were changed forever…
“We didn’t know anything about Duchenne and felt isolated that there was no one we could turn to… CureDuchenne has hosted several workshops that have been invaluable in providing knowledge on the care, treatment and future of our son, but, even more importantly, has enabled us to spend time with other Duchenne families…”
Duchenne is a devastating muscle disease.
It’s the most common and severe form of muscular dystrophy. Those affected with Duchenne lose their ability to walk, feed themselves, breath independently and succumb to heart failure. But there’s hope through new pharmacological and gene-based therapies. You can help make a difference.
Data show for the first time a systemic therapeutic effect in DMD dogs using a rAAV2/8 micro-dystrophin gene therapy approach without immunosuppressive treatment CAMBRIDGE, Mass., July 27, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today […]
CURE DUCHENNE NEWS
NEWPORT BEACH, Calif., June 15, 2017 – RASRx announced today that the United States Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) for its compound RASRx1902 for the treatment of Duchenne Muscular Dystrophy. RASRx1902 is an oral therapy that has shown positive effects on muscle function in animal models of Duchenne. […]