Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies ...

Biogen’s salanersen is now heading into three Phase III trials in SMA.

New Phase 1b data support the safety and effectiveness of salanersen over one year in children with SMA who had the potential for improvement due to suboptimal clinical status with prior gene ...

Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA)New Phase 1b data further illustrate potential of salanersen in SMA, ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

US biotech major Biogen has presented additional results from the Phase Ib study of salanersen, an investigational novel ...

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Biogen offers a financial assistance program for its treatment Spinraza but there’s a catch: patients must sign away their protected health information to the company, reports the Waco Tribune-Herald.

Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down ...

New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today called the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), developed ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said in a statement ...