Medical Xpress

What you should know about spinal muscular atrophy

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...
BIOENGINEER

Telomere Gene NHP2 Linked to Nerve-Driven Muscle Wasting in Rare Family Study

A Norwegian family study reveals that muscle wasting in NHP2-related dyskeratosis congenita is driven by neurogenic ...

Scholar Rock Announces FDA Approval of ISEMBYLD™ (apitegromab-mstn), the First and Only Muscle-Targeted Treatment for Children and Adults with Spinal Muscular …

CAMBRIDGE, Mass.-- (BUSINESS WIRE)--Sep 11, 2026-- ...
BioSpace

On the move: 4 rare disease companies hiring now

Biotechs developing therapeutics to treat rare diseases include two that received FDA approvals this year for drugs to treat ...

A camera combined with AI could help further diagnosis of movement disorders like spinal muscular atrophy in newborns

Detecting impaired motor function in infants using AI-driven motion capture is producing promising results. A French research ...
MarketScreener

Scholar Rock Receives FDA Approval For ISEMBYLD Apitegromab-Mstn For Treatment Of Spinal Muscular Atrophy

Scholar Rock announced that the FDA has approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in adults and children two years of age and older who are currently ...
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Scholar Rock gets first FDA nod for rare muscle-wasting disease (September 11)

(Corrects paragraph 9 of September 11 story to remove reference to the newly approved drug competing directly with treatments from Biogen, Novartis and Roche) By Sneha S K and Christy Santhosh Sept 11 ...