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.

Endoplasmic Reticulum and/or Mitochondrial-Dependent Neuronal Degeneration in ALS

June 14, 2022

Given the importance of mitochondrial and endoplasmic reticulum function in ALS, the disruption of intracellular mitochondria-endoplasmic reticulum contacts presents yet another avenue for neuronal degeneration—perhaps the primary point of underlying dysfunction.

FDA Approves Vutrisiran to Treat hATTR Amyloidosis

June 14, 2022

The approval was based on results from the phase 3 HELIOS-A study, which showed that vutrisiran met its primary end point of change in modified Neuropathy Impairment Score + 7 over a 9-month treatment period.

NeurologyLive® Brain Games: June 12, 2022

June 12, 2022

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is the history of the American Headache Society.

FibroGen Completes Enrollment in LELANTOS-2 Trial of Pamrevlumab in Ambulatory DMD

June 09, 2022

The trial is set to read out topline data in the second half of 2023 after completing the target enrollment of 73 patients with Duchenne muscular dystrophy aged 6 to 12 years. The connective tissue growth factor inhibitor has been granted orphan drug, fast track, and rare pediatric disease designations.