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

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 ...

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 ...

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

Add Yahoo as a preferred source to see more of our stories on Google. Biogen said the trial touted the once-yearly dosing potential of the ASO. Credit: Tada Images / Shutterstock.com (Tada Images / ...

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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 ...

"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...

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.

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 ...