Over the past few years, a number of novel treatments for neuromuscular disorders were approved and are now available to patients in the clinic, showcasing the drastic evolution of translational research in the field. Additionally, several other clinical trials are in the pipeline for various conditions.

In this episode, Sabrina Paganoni, MD, PhD, Massachusetts General Hospital, Harvard Medical School, Boston, MA, James F. Howard, Jr., MD, FAAN, University of North Carolina School of Medicine, Chapel Hill, NC, and Jerry Mendell, MD, FAAN, Nationwide Children’s Hospital & Ohio State University, Columbus, OH, discuss key clinical trials in neuromuscular disorders that read out over the last year. Our panel of experts will review the results of the CENTAUR trials (NCT03127514, NCT03488524) of AMX35 for amyotrophic lateral sclerosis (ALS), the Phase III ADAPT trial (NCT03669588) evaluating efgartigimod in patients with generalized myasthenia gravis (MG), and the START trial (NCT02122952) long-term follow-up of onasemnogene abeparvovec gene therapy in spinal muscular atrophy (SMA).