Neuromuscular Disease News

In this video perspective, Sabrina Paganoni, MD, PhD, discusses her presentation of results from the CENTAUR trial in ALS at the American Academy of Neurology annual meeting, which is being held virtually.

Avalglucosidase alfa provided “substantial improvements in clinically meaningful outcome measures” and a more favorable safety profile for late-onset Pompe disease compared with Lumizyme, according to findings from the phase 3 COMET trial.

Researchers developed a new 14-item patient-reported outcome measure for assessing quality of life in men and boys with Duchenne muscular dystrophy, according to results published in Neurology.

AMX0035, an investigational neuroprotective therapy, significantly lowered the long-term risk for death or need for permanent assisted ventilation, as well as risk for first hospitalization, according to a press release.

Emflaza, a corticosteroid, improved or stabilized disease progression in most patients with Duchenne muscular dystrophy or Becker muscular dystrophy who switched from prednisone, according to a press release.

Evrysdi, a survival motor neuron 2-directed RNA splicing modifier, both sustained and improved motor function at 24 months in children and adults with type 2 or type 3 spinal muscular atrophy, according to results from the SUNFISH trial.

The FDA announced that it has granted accelerated approval for Amondys 45 injections as a treatment for a rare form of Duchenne muscular dystrophy, according to a press release.

A research collaboration will investigate the use of lipid nanoparticles to deliver therapies for Charcot-Marie-Tooth disease to the peripheral nervous system, according to a press release from the CMT Research Foundation.

A rapid-tapering prednisone regimen was found to be feasible, well-tolerated and associated with good outcomes in patients with moderate to severe generalized myasthenia gravis, according to findings published in JAMA Neurology.

Mitsubishi Tanabe Pharma America and Aquestive Therapeutics announced a licensing and supply deal for U.S. rights to commercialize an oral film formulation of riluzole for the treatment of ALS, according to a press release.