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.
NIH Cognitive Battery Effectively Screens for Cognitive Issues in Duchenne Muscular Dystrophy
September 19th 2019A prospective, cross-sectional assessment suggests that patients with DMD can be adequately assessed for cognition in a brief period of time using the NIHTB‐CB, and additionally suggested a cognitive vulnerability in mothers who were carriers of DMD mutation.
Nusinersen's Long-Term Success in SMA Lends Support to Higher Dose Trial
September 19th 2019Further analysis of the SHINE trial suggest nusinersen is linked to 6-year maintained improvement in patients with SMA. Biogen has announced a new global trial of a 50 mg loading dose in a broader cohort.
Zilucoplan Gets Orphan Designation in Myasthenia Gravis Ahead of Phase 3 Trial
September 10th 2019Ra Pharmaceuticals’ macrocyclic peptide inhibitor of complement component 5 is expected to enter phase 3 of its development in the latter half of 2019 after success in a phase 2 trial of its potential in myasthenia gravis.
Serum Neurofilament Light Promising in ALS as Prognostic Indicator of Death
August 29th 2019Serum NfL levels, at as early as the time of diagnosis, were a strong and independent predictor of death in patients with amyotrophic lateral sclerosis, with the study results expanding on the findings of prior literature.
The Symbiotic Relationship of Artificial Intelligence and Neurology
August 18th 2019The associate chief of the MS division and professor of neurology at the University of Pennsylvania described the relationship between artificial intelligence and medicine, and how he sees it evolving in the future.
NORD and C-Path Announce Rare Diseases Data and Analytics Platform
August 8th 2019The Rare Disease Cures Accelerator-Data and Analytics Platform, aimed at accelerating the development of new therapies, will launch at a joint meeting between the Critical Path Institute and the National Organization for Rare Disorders on Tuesday, September 17 in Bethesda, Maryland.
Can Telemedicine Make a Difference in Neurology Subspecialties?
August 5th 2019The staff neurologist at Cleveland Clinic’s Mellen Center for MS shared her insight into the use of telemedicine in an outpatient setting across a number of subspecialties in neurology and how it can supplement care going forward.
FDA Cites Active Ingredient Issue With Riluzole For Treatment of Amyotrophic Lateral Sclerosis
July 22nd 2019The complete response letter cited issues with an active pharmaceutical ingredient used in the Biohaven 2017 bioequivalence study that was manufactured between 2014 and 2016 in an Apotex facility.
Future of Neurology Congress Will Offer Expert Insights on Hot Topics
July 15th 2019The International Congress on the Future of Neurology will take place this Fall in New York City, converging experts in neurology to discuss the latest data and best practices to better inform clinical decision-making.
Identifying Myasthenia Gravis Earlier in Patients Who Fail to Seek Treatment
July 2nd 2019The clinical assistant professor of neurology and neurosurgery at the University of Texas discussed the need to identify men who may be at risk for myasthenia gravis despite a lack of willingness to see their physician.
Investigational DMD Gene Therapy Reports Positive Preliminary Results in Phase 1b
July 1st 2019PF-06939926 is a recombinant adenoassociated virus serotype 9 (AAV9) capsid carrying mini-dystrophin, a shortened version of the human dystrophin gene, under the control of a muscle-specific promoter.
Addressing Serious Neurologic Conditions in Men
June 28th 2019The clinical assistant professor of neurology and neurosurgery at the University of Texas spoke about the staggering number of men who forgo seeing physicians for serious conditions and how the clinical community can help address the challenge.