Capricor Therapeutics has shared positive 18-month results from its ongoing HOPE-2 open label extension (OLE) study of CAP-1002 in patients with later-stage Duchenne muscular dystrophy.
Continue readingREGENXBIO: Phase I/II AFFINITY DUCHENNE™ trial of RGX-202
REGENXBIO has initiated Phase I/II of AFFINITY DUCHENNE™, a gene therapy clinical trial of RGX-202 in the United States.
Continue readingPepGen: Duchenne Community Newsletter
PepGen has provided an end of year update for the Duchenne muscular dystrophy community. Please see their newsletter from Q4 2022.
Continue readingSanthera and ReveraGen: FDA Acceptance of New Drug Application
Santhera Pharmaceuticals and ReveraGen BioPharma, Inc have announced that the U.S. Food and Drug Administration (FDA) has
accepted the new drug application (NDA) for vamorolone for the treatment of Duchenne muscular
dystrophy.
Sarepta Therapeutics: Submits Biologics License Application for SRP-9001
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.
Continue readingDefeat Duchenne Canada: Sponsored Canadian Clinical Trial Reaches New Milestone
Defeat Duchenne Canada is delighted to announce that the sponsored Canadian clinical trial of vamorolone has reached a key milestone (trial midpoint) of opening higher dose treatment groups to new enrollment.
Continue readingPepGen: Positive Data from Phase 1 Trial of PGN-EDO51
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.
Continue readingRoche: Duchenne Research Interview Request
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.
Continue readingEdgewise Therapeutics: Positive 4-Month Interim Results from ARCH (EDG-5506)
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).
Continue readingEdgewise Therapeutics: FDA Authorization for Phase 2 Clinical Trial of EDG-5506
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.
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