As an early funder of Capricor Therapeutics, CureDuchenne is pleased to share that the FDA has set the PDUFA target action date for August 22, 2026 as the deadline to […]
NS Pharma, Inc. presented 4.5-year safety and efficacy data based on the open-label extension study of brogidirsen (NS-089/NCNP-02) an antisense oligonucleotide for DMD patients with mutations amenable to skipping exon […]
Santhera Pharmaceuticals announced results from the ongoing Guardian study in Duchenne, and showed that AGAMREE had comparable effectiveness compared to historical cohorts taking prednisone or deflazacort, based on time to […]
As an early investor in Dyne Therapeutics, CureDuchenne is happy to share that new analyses out to 24 months showed improvement in heart and lung function with z-rostudirsen compared to […]
Roche has decided to stop recruitment for their Phase 2 SHIELD DMD study of satralizumab in Duchenne, citing the decision was not due to any new efficacy or safety issues, but rather […]
We are disappointed to report that PTC Therapeutics has withdrawn its New Drug Application for Translarna™ (ataluren) for the treatment of nonsense mutation DMD after feedback from the FDA that the […]
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Precision BioSciences announced that the U.S. FDA has cleared its Investigational New Drug (IND) application for a gene editing therapy for Duchenne muscular dystrophy. This clearance allows the company to move […]
CureDuchenne Invests in Satellos to Advance a Mutation-Independent Duchenne TherapyLink to press release CureDuchenne is pleased to announce that it has participated in the Satellos Bioscience public offering, supporting the […]
