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.
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REGENXBIO has initiated Phase I/II of AFFINITY DUCHENNE™, a gene therapy clinical trial of RGX-202 in the United States.
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PepGen has provided an end of year update for the Duchenne muscular dystrophy community. Please see their newsletter from Q4 2022.
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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.
Defeat Duchenne Canada’s National Vice-President of Research, Advocacy and Education, Nicola Worsfold, attended the 27th Annual International Congress of the World Muscle Society and has provided a summary for the Canadian Duchenne muscular dystrophy community.
Continue readingSanthera Pharmaceuticals has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD).
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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.
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Solid Bioscience has announced the acquisition of AavantiBio, the pause of their first-generation gene transfer therapy candidate SGT-001, and the prioritization of SGT-003, their next-generation adeno-associated virus (AAV) gene transfer therapy candidate.
Continue readingDefeat 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 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.
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