Solid Biospace Gene Therapy SGT-003, an investigational microdystrophin gene therapy for Duchenne, continues to demonstrate an encouraging safety profile and has been generally well tolerated in 40 participants treated to […]
ITF shared a community letter and presented new data, including long-term safety observations based on data from the company’s ongoing open-label extension study in patients with Duchenne muscular dystrophy treated with […]
REGENXBIO GENE THERAPY RGX-202, an investigational gene therapy for Duchenne, continues to show a favorable safety profile with no serious adverse events, no liver injury signals, and reductions in key […]
Edgewise Therapeutics, a CureDuchenne funded company, today announced positive long-term Sevasemten data that demonstrated sustained functional stabilization in Becker Muscular Dystrophy (BMD) patients through 3.5 years of treatment. This result […]
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 […]
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We are disappointed to report that PTC Therapeutics has withdrawn its New Drug Application for Translarna™ (ataluren) for the treatment of nonsense mutation DMD after feedback from the FDA that the […]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 […]
