Santhera: Marketing Authorization Application to the European Medicines Agency for Vamorolone

Sarepta Therapeutics has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of SRP-9001, an investigational gene therapy for Duchenne muscular dystrophy.

PepGen has announced the results from their completed Phase 1 healthy normal volunteer (HNV) trial of PGN-EDO51, the company’s lead exon skipping product for Duchenne muscular dystrophy.

Roche is now recruiting caregivers and patients who are able to offer their perspectives on completing the North Star Ambulatory Assessment (NSAA) or Performance of Upper Limb (PUL) as part of a new research study.

Edgewise Therapeutics has announced positive 4-month interim results from the ARCH open label study of EDG-5506 in adults with Becker muscular dystrophy (BMD).

The U.S. Food and Drug Administration (FDA) has authorized a clinical trial of EDG-5506, an investigational orally administered small molecule myosin modulator designed to protect injury-susceptible fast skeletal muscle fibers in Duchenne and Becker muscular dystrophy.

The U.S. Food and Drug Administration (FDA) has removed the clinical hold on SRP-5051 (vesleteplirsen), Sarepta’s investigational peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) to treat patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.

Dyne Therapeutics has achieved dosing the first patient in their Phase 1/2 clinical trial, DELIVER, evaluating DYNE-251 for the treatment of Duchenne muscular dystrophy mutations amenable to exon 51 skipping.