Neuromuscular

The NeurologyLive neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.

ADAPT Trial of Efgartigimod and its Potential in gMG: James F. Howard, MD

June 16, 2021

The Distinguished Professor of Neuromuscular Disease at UNC School of Medicine discussed the findings of the ADAPT trial and the promise efgartigimod has shown to be an effective agent for generalized myasthenia gravis.

Myostatin Inhibition in Amyotrophic Lateral Sclerosis

June 16, 2021

ALS has only recently been associated with causative or disease-modifying mutations in 20 genes that encode proteins with diverse functions, with promising therapeutic targets including proteins in pathways that regulate protein homeostasis.