The US Food and Drug Administration (FDA) has approved the antisense oligonucleotide casimersen (Amondys 45, Sarepta Therapeutics) injection for the treatment of patients with Duchenne muscular ...

Shares of Dyne Therapeutics DYN rose 10% on Wednesday after the company announced the initiation of the phase III FORZETTO ...

Primary endpoint is rise from floor (RFF) velocity with multiple secondary endpoints to assess muscle and pulmonary function ...

Duchenne muscular dystrophy (DMD) is a genetic condition caused by a change, or mutation, in the DMD gene. This gene normally tells the body how to make dystrophin, a protein that helps protect ...

CureDuchenne, a global leader in funding and advancing research for Duchenne muscular dystrophy, today announced a second investment into Tevard Biosciences to support the advancement of the company's ...

Gemma Biotherapeutics ("GEMMABio"), a clinical‑stage, global, genetic medicines company, today announced the presentation of preclinical data supporting candidate declaration for GB703, a novel, ...

- PBGENE-DMD is a first-in-class in vivo gene editing approach for the majority of Duchenne Muscular Dystrophy patients impacted by dystrophin mutations in the most common ‘hot spot’ region between ...

Dyne Therapeutics reports positive long-term data for DYNE-251 in Duchenne muscular dystrophy, aiming for U.S. accelerated approval in 2026. Dyne Therapeutics announced new long-term clinical data ...

Certain genetic mutations in patients with DMD influence the timing of ambulation loss, with exon 44 skipping, exons 3 through 7 deletions, and exon 2 duplications linked to delayed loss. The study's ...

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing ...