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.
The Future of Genetic Testing in ALS: Calaneet Balas, MSc, MBA
March 29th 2022The president and chief executive officer of The ALS Association provided insight on the availability of genetic testing for ALS and the organization’s efforts to increase access for the future. [WATCH TIME: 2 minutes]
Long-Term Functional Abnormalities Common in Severe COVID-19 Cases
March 28th 2022Abnormal scores on cognitive testing persisted in 50% of patients without a pre-COVID history of cognitive abnormalities, irrespective of the presence or absence of a neurological complication during hospitalization.
Unraveling the Complexities of Cerebral Palsy and Building Hope for a Brighter Future
March 24th 2022During Cerebral Palsy Awareness Month, leaders in the field expressed their love for the CP community, the progress made in managing the disorders, and the steps needed to advance care into a realm never before seen.
Gaining a Clearer Picture of Gene Therapy in Neuromuscular Disease: Carsten G. Bönnemann, MD
March 23rd 2022The senior investigator in the Neuromuscular and Neurogenetic Disorders of Childhood Section at NINDS shared his perspective on the data coming out of neuromuscular research and the promise of genetic medicine. [WATCH TIME: 2 minutes]
Subcutaneous Efgartigimod Shows Noninferiority to IV Formulation in Generalized Myasthenia Gravis
March 23rd 2022Topline data from the phase 3 ADAPT-SC study (NCT04735432) suggest that the subcutaneous formulation of the Argenx product was statistically noninferior to the recently approved IV formulation, efgartigimod afla-fcab (Vyvgart).
Improving and Expanding Access to ALS Treatment Options: Calaneet Balas, MSc, MBA
March 23rd 2022The president and chief executive officer of The ALS Association provided commentary on the most pertinent issues with managing patients with ALS and the ways to improve current treatment standards. [WATCH TIME: 4 minutes]
The Need for Participation in Neuromuscular Disease Clinical Trials: Donald S. Wood, PhD
March 22nd 2022The president and CEO of the Muscular Dystrophy Association spoke about the need for increased participation in neuromuscular disorder clinical trials and the rapid therapeutic progress the field is seeing. [WATCH TIME: 4 minutes]
BBP-418 Demonstrates Significant Impact on Root Cause of Limb-Girdle Muscular Dystrophy Type 2i
March 22nd 2022Eleven of the 12 individuals demonstrated at least a 50% reduction in creatine kinase, with 75% of participants reaching twice the normal range, suggesting a reduction in muscle breakdown.
Successes and Challenges in the New Era of Genetic Medicine: Nicholas E. Johnson, MD, MSci
March 20th 2022The division chief of neuromuscular disorders and vice-chair of research at Virginia Commonwealth University spoke to the ongoing success in the development of gene-mediated therapies and the challenges that come along with treating rare neuromuscular disease. [WATCH TIME: 3 minutes]
NeurologyLive® Brain Games: March 20, 2022
March 20th 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 optic neuritis and neuromyelitis optica spectrum disorder (NMOSD).
Zolgensma Reinforces Efficacy in SPR1NT Results of Patients With SMA and 3 Copies of SMN2
March 19th 2022The gene therapy from Novartis showed that it was both efficacious and well-tolerated in presymptomatic patients with SMA with 3 copies of SMN2, with no patients experiencing treatment-related serious adverse events.
Understanding the Newest Therapeutic Advances in Neuromuscular Diseases
March 17th 2022Jinsy Andrews, MD, MSc, FAAN, offered her perspective on the ongoing therapeutic boom in neuromuscular disease and some of the critical needs for physicians to keep up with a rapidly progressing treatment paradigm.