Neuromuscular Disease News

Biotechnology company bluebird bio announced that an FDA advisory committee has unanimously endorsed its gene therapy treatment for early active cerebral adrenoleukodystrophy.

A sensor-equipped computer program was nearly 80% effective in identifying and counting arm movements in patients undergoing stroke rehabilitation, researchers from NYU Grossman School of Medicine announced.

CHARLOTTE, N.C. — The use of morning light treatment in patients with fibromyalgia helped stabilize their sleep and led to improvements in pain and physical function, Helen J. Burgess, PhD, said at the SLEEP 2022 meeting.

The FDA has extended its new drug application review of AMX0035, a combination of sodium phenylbutyrate and taurursodiol for the treatment of ALS, Amylyx Pharmaceuticals Inc. announced in a press release.

High efficacy and acceptability were demonstrated in both on-label and off-label medications for fibromyalgia, reinforcing the value of symptom-based treatment plans, according to a study published in JAMA Network Open.

Genentech announced that the FDA approved a label extension for Evrysdi to include infants younger than 2 months with spinal muscular atrophy.

Presence of axonal phosphorylated transactive response DNA-binding protein 43, or pTDP-43, accumulation in intramuscular nerve bundles is a likely biomarker for patients diagnosed with ALS, according to a study published in JAMA Neurology.

Wysa announced it has received breakthrough device designation from the FDA for its artificial intelligence-based digital mental health conversational agent for adults with chronic musculoskeletal pain, depression and anxiety.

The FDA has approved Radicava ORS oral suspension for the treatment of adults with ALS.

Stealth BioTherapeutics Corp. announced that the FDA has granted orphan drug designation to elamipretide for the treatment of Duchenne muscular dystrophy.