What is SMA? The rare genetic condition that Jesy Nelson’s twin daughters have - Former Little Mix star revealed her twin ...

The company discontinued developing emugrobart after two studies showed the drug didn’t achieve intended outcomes.

Indiana Orthopedic Institute is expanding patient access to expert musculoskeletal care to the southside area with the ...

The MDA's annual conference highlighted recent breakthroughs and the outlook for future care in the neuromuscular disease ...

In a letter to European patients, Roche said its decision to end development of “emugrobart” was based on the drug “not ...

The big pharma company will no longer progress emugrobart to late-stage trials in FSHD and SMA due to a lack of efficacy.

Mirecule, Inc., a leader in Antibody RNA Conjugate (ARC) therapeutics targeting rare neuromuscular diseases, today announced an expansion of its strategic collaboration with Sanofi. This collaboration ...

The pharmaceutical and biotech sectors have seen a flurry of executive movement this month, with leadership changes spanning ...

Mutations that cause spinal muscular atrophy (SMA) may lead to abnormalities in the development of cells in the spinal cord, a study found.

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

Data from BridgeBio Pharma’s Phase 3 FORTIFY study show that BBP-418 significantly increases levels of a key disease ...

The 2026 MDA Clinical & Scientific Conference Highlights Breakthroughs in Neuromuscular Research, Innovative Therapies, and ...