Rare Disease Report


According to data presented at the Annual Neuromuscular Translational Research Conference, researchers found that repeat dosing of CAP-1002 results in increased exercise performance in a disease model of Duchenne muscular dystrophy.
Pfizer announced that it has begun a Phase 1b clinical trial for PF-06939926, its mini-dystrophin gene therapy candidate, intended for boys with Duchenne muscular dystrophy.
Results from a study published in Nature Communications offer potential treatment targets for melorheostosis, often referred to as “dripping candle wax” bone disease.
Novartis and AveXis merge spells opportunity for SMA research.
Parents of children with the rare disease Congenital Muscular Dystrophy are spending the next seven weeks preparing for the 5th Annual Million Dollar Bike Ride, to be held in Philadelphia on May 20th.
A February 2018 Type C meeting between Sarepta Therapeutics, Inc. and the Division of Neurology Products of the U.S. FDA was held to solicit the Division’s guidance on the development pathway for Sarepta’s golodirsen (SRP-4053).
In this video, Stephen Spaulding, M.A., Director Of Care Services for the ALS Association Greater Philadelphia Chapter, discusses how certain science and drugs can apply to more than one patient population.
George Yohrling, Ph.D., Senior Director of Mission and Science at the HDSA discusses the challenges that come with working with patients with HD, and communicating with their family members and caregivers.
The U.S. FDA has granted Capricor Therapeutics RMAT designation for its lead investigational cell therapy for the treatment of Duchenne muscular dystrophy, CAP-1002.
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