Neuromuscular Disease News

For individuals with ALS, a daily dosing regimen of oral edaravone was nonsuperior to an FDA-approved staggered regimen of the drug plus placebo, according to a poster presented at AANEM.

Treatment with IV nipocalimab was safe and led to significant reductions in disease expression at 24 weeks in adolescents with generalized myasthenia gravis, according to data presented at AANEM.

An oral therapeutic significantly reduced the number of tumors in adults with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas, according to topline results from a phase 3 study.

IV administration of Uplizna was safe and shown to be more efficacious for those with generalized myasthenia gravis compared with placebo at week 26, according to a presentation from AANEM.

IV administration of fasudil at two separate doses was safe and well tolerated in a small cohort of individuals with suspected or confirmed ALS, according to new research published in The Lancet.

Biogen Inc. and Neomorph Inc. have announced a partnership geared toward research and development of molecular glue degraders to address protein accumulation related to Alzheimer’s disease and other neurologic conditions.

ADx NeuroSciences and Alamar Biosciences have announced a partnership to develop customized biomarker assays for companies that advance novel therapeutics for a range of neurological conditions.

A progressive neurodegenerative condition, ALS has no cure, with diagnosis often a result of protracted clinical evaluation that may take more than a year to accurately determine.

The European Medicines Agency has granted orphan drug designation to a small molecule intended to treat spinal and bulbar muscular atrophy, according to the manufacturer.