News

Sarepta to Ask FDA to Approve DMD Gene Therapy SRP-9001

Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant accelerated approval to the company’s experimental gene therapy SRP-9001 for people with Duchenne muscular dystrophy (DMD) who are able to walk. “We are delighted to confirm that based on the feedback we received…

First Participant With FSHD Dosed in Phase 3 Study of Losmapimod

The first participant has been dosed in the Phase 3 REACH clinical trial testing the safety and effectiveness of Fulcrum Therapeutics‘ experimental oral therapy losmapimod in people with facioscapulohumeral muscular dystrophy (FSHD). If results from the trial are positive, they’re expected to support applications for lospmapimod’s approval…

Group Seeks Addition of DMD to US Newborn Screening Panel

Using the results of a New York State pilot testing program as evidence, Parent Project Muscular Dystrophy (PPMD) submitted its proposal seeking the addition of Duchenne muscular dystrophy (DMD) to the nationwide newborn screening panel. The proposal nominates DMD for inclusion in the Recommended Uniform Screening Panel or RUSP…

HOPE 2 Study: CAP-1002 Still Improving Arm Function in DMD

The investigational cell therapy CAP-1002 continued to improve arm function in boys and young men with advanced Duchenne muscular dystrophy (DMD), according to the HOPE-2 open-label extension study. These additional upper limb improvements came after HOPE-2 participants were off CAP-1002 for a mean of about one year before…