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.
August 29th 2025
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 29, 2025.
Reactions, Ramifications, and Forward Progress of Newly Approved AMX0035 for ALS
October 1st 2022Justin Klee and Josh Cohen, cofounders and cochief operating officers, Amylyx Pharmaceuticals, answered questions related to the new approval of AMX0035 for ALS and how it changes the treatment landscape going forward.
Sleep Breathing Disorders Appear Common for Patients with Duchenne Muscular Dystrophy
September 30th 2022A questionnaire-based study from Saudi Arabia on sleep quality revealed that sleep-related disorders, such as obstructive sleep apnea, are relatively common in patients with Duchenne muscular dystrophy.
Open-Label Extension Data Highlight Tofersen’s Ability to Reduce SOD1 Protein in ALS
September 29th 2022Despite not improving clinical end points, treatment with tofersen was associated with reduced in the total concentration of SOD1 protein and neurofilament light chain, a marker of axonal injury.
Diagnosis of Myotonic Dystrophy Demonstrates an Association With Depression
September 29th 2022Findings from a cross-sectional survey revealed a significant association between depression and a diagnosis of myotonic dystrophy, and that symptoms impacting quality of life are more common in neuromuscular disorders.
New Treatments for Duchenne Muscular Dystrophy: Emma Ciafaloni, MD
September 27th 2022New Treatments for Duchenne Muscular Dystrophy: Emma Ciafaloni, MD The professor of Neurology and Pediatrics at University of Rochester Medical Center discusses the new and future treatments for Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]
Tele-Yoga Breathing for Respiratory Function Proves Feasibility in Amyotrophic Lateral Sclerosis
September 26th 2022A nonrandomized pilot study demonstrated high adherence rates and acceptability ratings with tele-yoga breathing in patients with ALS, proving that it is a safe and feasible practice.
Patients Receiving Efgartigimod Respond to COVID-19 Vaccination With IgG Antibodies
September 25th 2022In an investigation on the effect of treatment with efgartigimod on humoral immune responses to COVID-19 vaccination, the immunization resulted in antigen-specific IgG responses in most patients.
NeurologyLive® Brain Games: September 25, 2022
September 25th 2022Test 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 Association of Neuromuscular & Electrodiagnostic Medicine.
Unique Composition of CNM-Au8 to Treat MS and ALS: Michael Hotchkin
September 24th 2022The chief development officer at Clene Nanomedicine provided insight on why CNM-Au8, an investigational agent in development, has shown positive results across both multiple sclerosis and ALS. [WATCH TIME: 2 minutes]
Eplontersin Treatment Results in Reduction in Serum Transthyretin in Amyloidosis
September 23rd 2022Eplontersen treatment resulted in a significant reduction in transthyretin, neuropathy impairment, and improvement in quality of life in the phase 3 NEURO-TTRansform study of patients with hereditary transthyretin amyloidosis.