In November 2025, Biogen received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use recommending approval of a high dose nusinersen regimen for 5q ...

I’ve been reflecting on the disappointing outcomes and the reaction to Sarepta Therapeutics’ recently announced clinical ...

SMA expert Brian Lin, PhD, shares his thoughts on aging with SMA, treatment, advocacy, and supporting patients across their ...

Epirium Bio Inc. (Epirium), a clinical-stage biopharmaceutical company advancing medicines for neuromuscular and fibrotic diseases, presented Phase 1 data for its lead candidate, MF-300, at the ...

The recommendation is based on the Phase II/III DEVOTE study testing the higher dose in treatment-naïve patients and those already on the low dose.

Biogen on Monday said the positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use covers the high-dose regimen of Spinraza for the treatment of 5q SMA, ...

Positive CHMP opinion is based on data from the DEVOTE study which evaluated the high dose regimen of nusinersen in treatment-naive participants ...

A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients.

Scholar Rock Holding shares rose sharply Friday after the biopharmaceutical company gave a positive update on its efforts to win Food and Drug Administration approval of its proposed apitegromab ...

Combining the experimental and approved SMA therapies BIO101 and Spinraza in the lab was found to improve muscle function in ...

Biohaven Ltd. narrows focus to three late-stage clinical programs and implements cost-cutting to address heavy R&D spending.

Scholar Rock Holding Company ( SRRK) is a Boston-based, clinical-stage biotech that was founded in 2012, focusing on ...