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.
Newborn Screening for SMA: Clinical Recommendations and Considerations
October 11th 2024In this discussion, experts provide key considerations in the process of newborn screening for SMA, noting the need for developing local protocols and understanding the limitations of screening infants early in life.
FDA Removes Partial Hold for Myotonic Dystrophy Agent AOC 1001
October 10th 2024AOC 1001, also known as del-desiran, has shown promising results in improving muscle strength and reducing myotonia in patients with DM1, with patients even reporting better daily activities like hand grip strength.
Key Updates in SMA Diagnosis: What Clinicians Should Know
October 9th 2024In this episode, our panel of experts delves into the latest updates on spinal muscular atrophy diagnosis, focusing on the critical role of newborn screening, early intervention, and evolving classification methods for improved patient outcomes.
Promising Outcomes for Spinal Stenosis and Spondylolisthesis: An Alternative to Spinal Fusion
October 2nd 2024The Total Posterior Spine System offers a promising alternative to spinal fusion, preserving mobility and reducing strain on adjacent spinal levels in patients with spondylolisthesis and spinal stenosis.
NeurologyLive® Clinician of the Month Spotlight: Forum Kamdar, MD, PhD, FACC, FHFSA
September 30th 2024As part of our monthly clinician spotlight, NeurologyLive® highlighted neuromuscular-associated cardiomyopathy expert Forum Kamdar, MD, PhD, FACC, FHFSA, an advanced heart failure physician-scientist at the University of Minnesota.