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.
September 1st 2025
Here's some of what is coming soon to NeurologyLive® this week.
Nerve Ultrasound Is an Accurate Diagnostic Tool for Chronic Inflammatory Neuropathies
January 29th 2020New study data showed nerve ultrasound had a sensitivity of 97.4% and specificity of 78.9%, with an added value in the detection of treatment-responsive chronic inflammatory neuropathy of 21.1%.
Elampretide Fails to Meet Primary End Points in Primary Mitochondrial Myopathy Trial
December 27th 2019After promising results in prior phase 2 study, Stealth BioTherapeutics’ elampretide has failed to show change in the 6-minute walk test and Primary Mitochondrial Myopathy Symptom Assessment total fatigue score in the MMPOWER-3 trial.
The Road to Success in ALS: Details of the AMX0035 CENTAUR Trial
December 18th 2019CENTAUR trial principal investigator Sabrina Paganoni, MD, PhD, discussed details of the phase 2 trial that helped investigators have the greatest chance of seeing a measurable treatment effect in patients with ALS.
FDA Approves ALS Drug Riluzole Oral Suspension for Use in Feeding Tubes
December 14th 2019A survey by the ALS Association found that 26% of patients reported having a PEG tube, which are typically inserted to allow for continued administration of nutrition, fluids, and medications as the disease progresses.
FDA Approves Golodirsen for DMD Amenable to Exon 53 Skipping
December 13th 2019After receiving expedited review after receiving a Complete Response Letter in August, the therapy was approved based on data showing a statistically significant increase in dystrophin production in skeletal muscle.