Neuromuscular Disease News

The FDA has approved Relyvrio, an oral, fixed-dose combination therapy for the treatment of adults with ALS, Amylyx Pharmaceuticals Inc. announced in a press release.

Results of a recently released poll revealed that 77% of small business owners said they have prioritized mental health since the onset of the COVID-19 pandemic.

A single, 500 mg dose of rituximab was associated with greater probability of minimal myasthenia gravis manifestations and reduced the need for rescue medications compared with placebo, researchers reported in JAMA Neurology.

A phase 3 trial that evaluated the safety and efficacy of the intrathecally administered antisense oligonucleotide tofersen reduced concentrations of superoxide dismutase 1 protein in patients with ALS who had mutations in the gene.

Acupuncture was shown to be an effective treatment for anxiety in patients with Parkinson’s disease, researchers reported in JAMA Network Open.

The FDA and NIH announced the launch of the Critical Path for Rare Neurodegenerative Diseases, a public-private partnership aimed to advance the understanding of neurodegenerative diseases and the development of treatments.

The FDA granted accelerated approval to bluebird bio Inc for elivaldogene autotemcel, its gene therapy that aims to slow progression of neurologic dysfunction in boys aged 4 to 17 with early, active cerebral adrenoleukodystrophy, or CALD.

The FDA has accepted a new drug application from Acadia Pharmaceuticals Inc. for trofinetide, a synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat core symptoms of Rett syndrome.

Amylyx Pharmaceuticals announced that the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted to support approval of the company’s oral, fixed-dose therapeutic AMX0035 for the treatment of ALS.

Edgewise Therapeutics Inc. announced positive 4-month interim results from the ongoing ARCH study of investigational, orally administered EDG-5506 for the treatment of Becker muscular dystrophy in adults.