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 Approves Medtronic’s Inceptiv Closed-Loop Spinal Cord Stimulator
Published: April 30th 2024 | Updated: May 2nd 2024The FDA approval of Medtronic’s spinal cord stimulator makes it the first on the market to offer a closed-loop feature that senses biological signals along the spinal cord and automatically adjusts stimulation in real time for chronic pain.
Promise of Neuroprotection in Neurodegenerative Diseases Through HGF Compounds: Kevin Church, PhD
April 24th 2024The chief scientific officer at Athira talked about the potential of hepatocyte growth factor compounds in promoting neurotrophic signaling and providing broad neuroprotection across neurodegenerative diseases. [WATCH TIME: 6 minutes]
Examining Promising Phase 2b PARADIGM Findings of ALS Agent PrimeC: Merit Cudkowicz, MD, MSc
April 24th 2024The chair of neurology at Massachusetts General Hospital talked about results from the phase 2b PARADIGM trial assessing PrimeC, an ALS combination agent that consists of ciprofloxacin and celecoxib. [WATCH TIME: 4 minutes]
AMX0035 Improves Pancreatic Function and Glycemic Control in Wolfram Syndrome, Phase 2 Results Show
April 11th 2024All 8 participants met prespecified responder criteria demonstrating either improvement or stabilization of disease according to both the Patient Reported Global Impression of Change and the Clinician Reported Global Impression of Change scales.
FDA Agrees to New Specialized Protocol for Phase 3b Study of ALS Agent NurOwn
April 10th 2024The new double-blind, placebo-controlled study includes approximately 200 patients with ALS who will undergo a single bone marrow aspiration procedure to procure the mesenchymal stem cells that will be used to manufacture each participant’s NurOwn treatment for the trial.
Current Roadblocks Toward Expanding Gene Therapy Across Pediatric Neurology: Shafali Jeste, MD
April 10th 2024The chief of neurology and co-director of the Neurological Institute at the Children’s Hospital Los Angeles described some of the major resource and policy-level barriers for gene therapy in pediatric neurological conditions. [WATCH TIME: 6 minutes]