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 1st 2025
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 1, 2025.
Mechanistic Effectiveness of Combined Approach With Nusinersen and Zolgensma: Crystal Proud, MD
July 13th 2023The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, provided background on findings from the phase 4 RESPOND study, and the potential of combining therapeutic approaches in SMA. [WATCH TIME: 4 minutes]
Zilucoplan Demonstrates Consistent Efficacy in Phase 3 RAISE Study of Generalized Myasthenia Gravis
July 12th 2023Nearly half of patients treated with zilucoplan were considered responders on Myasthenia Gravis Activities of Daily Living (MG-ADL) at the first week, suggesting a rapid onset of efficacy.
NeuroVoices: Crystal Proud, MD, on Improving SMA Outcomes Through a Combination Approach
July 12th 2023The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, provided perspective on the phase 4 RESPOND study assessing nusinersen in individuals with SMA who already received gene therapy.
Revolutionizing Care for Myasthenia Gravis With the Shift to Subcutaneous Therapy
July 4th 2023Vera Bril, MD, FRCPC, lead investigator of the MycarinG study, shared her reactions to recent approval of rozanolixizumab-noli, the first approved therapy for both subtypes of generalized myasthenia gravis in adults.
Gene Editing as a Promising Treatment Approach for Duchenne Muscular Dystrophy: Walter Strapps, PhD
June 30th 2023The chief scientific officer at Liberate Bio discussed how through the advancement of nonviral delivery technologies and nucleic acid therapeutics, genetic medicines can potentially bring relief to patients with Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]
Pegcetacoplan to be Discontinued in ALS Following Disappointing Phase 2 Results
June 24th 2023In a trial of 250 adults with sporadic ALS, pegcetacoplan failed to meet its primary end point of change in Combined Assessment of Function and Survival rank scores relative to placebo after a 52-week stretch.