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.
Counseling Patients About Treatment Outcomes in Spinal Muscular Atrophy
Panelists delve into strategies for engaging in conversations with patients about defining and identifying successful therapy outcomes, providing guidance on how to effectively communicate and establish realistic expectations for treatment success in the context of spinal muscular atrophy (SMA).
Determining Treatment Success in Spinal Muscular Atrophy
Panelists share their approaches to counseling adult patients with spinal muscular atrophy (SMA) about treatment expectations.
Switching Treatments in Spinal Muscular Atrophy
Key opinion leaders share their strategies for discussing potential changes in therapy and transitioning between treatments with patients, emphasizing the importance of reassuring patients that such changes are intended to provide long-term benefits for managing their spinal muscular atrophy.
Strength Training Provides Several Noted Benefits to Women With Myotonic Dystrophy Type 1
July 2nd 2024Following the 12-week program, participants reported numerous physical and neuropsychiatric benefits in their daily living, such as increased energy level, better mood, and improvement in balance.
Factors Guiding Treatment Selection in Adult Spinal Muscular Atrophy
Panelists explore critical considerations when choosing a treatment approach for treatment-naïve adult patients with spinal muscular atrophy (SMA), taking into account factors such as disease severity, patient preferences, and potential treatment outcomes.
Treatments Approved for Treatment of Spinal Muscular Atrophy
Key opinion leaders (KOLs) examine the mechanisms of action and other pertinent information regarding FDA-approved treatments for spinal muscular atrophy (SMA), sharing their perspectives on these therapies with a particular focus on the various modes of administration.
Previewing ALS Association’s Inaugural Nexus Conference: John Novak, MD, MS
June 28th 2024The director of the OhioHealth ALS Clinic and vice chair of the ALS Association Care Services Committee discussed the upcoming ALS Nexus Conference and how themes of the event align with the direction of clinical care. [WATCH TIME: 3 minutes]
Unseen Battles: Advocacy and Community in the Face of Myasthenia Gravis
June 28th 2024Anaya, a young woman with myasthenia gravis, discusses her challenges and triumphs living with a rare disease. She shares her personal story of advocacy and community, finding strength in the disease's impact on her independence and identity.