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.
July 30th 2025
Capricor Therapeutics navigates FDA challenges for deramiocel, aiming to advance treatment for DMD-related cardiomyopathy with promising trial data.
FDA Grants Rare Pediatric Disease Designation to DMD Gene Therapy Candidate SGT-003
May 16th 2024Solid Biosciences is expecting to provide initial safety data on SGT-003 from the first couple of pediatric patients with Duchenne muscular dystrophy enrolled in the phase 1/2 INSPIRE Duchenne trial in mid-2024.
Adding New Mechanistic Agents to SMA Treatment: the Phase 3 RESILIENT Study
May 13th 2024Lindsey Lee Lair, MD, a neurologist and vice president of clinical development at Biohaven Pharmaceuticals, provided insight on an ongoing pivotal trial assessing the impacts of taldefgrobep alfa, a myostatin inhibitor, in patients with SMA.
CSF Anti-DAGLA Autoantibodies Identified as Potential Biomarker of Rapidly Progressive Cerebellitis
May 11th 2024A recent study recognized anti-DAGLA autoantibodies in the cerebrospinal fluid of a small group of patients, suggesting that it could be a potential biomarker for diagnosing cerebellar ataxia.
NeuroVoices: Kayla Scippa, on Recognizing Unmet Need in Chronic Demyelinating Polyneuropathy
Published: May 8th 2024 | Updated: May 8th 2024The associate director, Patient Reported Outcomes, Johnson & Johnson, provided clarity on a poster presentation from AAN 2024 examining the most critical aspects to CIDP disease severity.
Understanding the Therapeutic Potential of Nipocalimab: Katie Abouzahr, MD
Published: May 6th 2024 | Updated: May 6th 2024The vice president, Autoantibody Portfolio and Maternal Fetal Immunology Disease Area Leader, Johnson & Johnson, provided perspective on the unique molecular structure of nipocalimab seen across nonclinical and clinical studies. [WATCH TIME: 3 minutes]
Addressing Potential Causes of Burnout Through Productive Solutions: Neil Busis, MD
Published: May 3rd 2024 | Updated: May 3rd 2024The associate chair for technology and innovation at the NYU Grossman School of Medicine provided commentary on a study from AAN 2024 that examined the driving factors behind burnout in neurology departments. [WATCH TIME: 4 minutes]