Neuromuscular Disease News

DENVER — Losmapimod treatment for those with facioscapulohumeral muscular dystrophy led to no serious adverse events across three clinical trials, according to a poster at the American Academy of Neurology annual meeting.

The FDA has granted regenerative medicine advanced therapy designation to an intrathecally delivered AAV9 gene transfer therapy to treat younger individuals with Rett syndrome, according to the manufacturer.

DENVER — A panel of four experts discussed different aspects of the ways brain health and climate change are linked at the American Academy of Neurology annual meeting.

DENVER — Uncontrolled generalized myasthenia negatively affects patients’ mental health, which in turn negatively impacts their physical symptoms, according to a poster presentation at the American Academy of Neurology annual meeting.

Wearable accelerometers may be a reliable tool to track disease progression in patients with amyotrophic lateral sclerosis, researchers wrote in The Lancet.

The FDA has approved Ingrezza sprinkle capsules to treat adults with Huntington’s disease chorea or tardive dyskinesia, according to a press release from the manufacturer.

DENVER — A combination therapeutic for amyotrophic lateral sclerosis was nonsuperior to placebo over 48 weeks in a phase 3 clinical trial, according to a presenter at the American Academy of Neurology annual meeting.

DENVER — Four studies presented at the American Academy of Neurology annual meeting show improvements in patients with spinocerebellar ataxia who take troriluzole.

The FDA has cleared an investigational new drug application for a small molecule therapeutic to treat those with Fragile X syndrome, according to the manufacturer.

DENVER — Treatment with IV efgartigimod improved functionality for individuals with acetylcholine receptor-positive generalized myasthenia gravis, according to a poster from the American Academy of Neurology annual meeting.