Neuromuscular Disease News

Riluzole did not improve clinical or radiological outcomes in patients with moderate spinocerebellar ataxia type 2, according to study results published in Lancet Neurology.

Vamorolone maintained muscle strength and function, as well as improved height velocity, compared with glucocorticoid therapy among those with Duchenne muscular dystrophy, according to data published in JAMA Network Open.

Healio’s most-viewed neurology stories in January highlight several FDA developments, a Medicare proposal to limit Aduhelm coverage and a study on the role of Epstein-Barr virus infection in MS.

The long-term use of IV edaravone in patients with ALS, although tolerated, did not produce significant improvement compared with standard therapies alone, according to a study published in JAMA Neurology.

The FDA has granted priority review to PTC Therapeutics Inc. for a supplemental new drug application regarding Evrysdi, according to a company release.

Epstein-Barr virus infection, which causes mononucleosis, may represent a primary cause of MS, according to results of a longitudinal analysis published in Science.

PharmaTher Holdings Ltd., a specialty ketamine prescription-based product development company, announced the FDA has accepted an investigational new drug application for proceeding with a phase 2 clinical trial to examine ketamine for ALS.

Clinical-stage biopharmaceutical company Immunovant Inc. announced it has reached agreement with the FDA to initiate a phase 3 study of batoclimab in patients with myasthenia gravis in the first half of the year.

The FDA has accepted a new drug application, or NDA, for a combination of sodium phenylbutyrate and taurursodiol intended to treat ALS, Amylyx Pharmaceuticals Inc. announced in a press release.

Individuals with pediatric-onset MS may exhibit disturbances in their gut microbiome, according to study results published in Neurology.