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 29th 2025
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 29, 2025.
MDA 2022 Highlights the Therapeutic Advances for Neuromuscular Diseases: Donald S. Wood, PhD
March 17th 2022The president and CEO of the Muscular Dystrophy Association spoke about the highlights of this year’s annual meeting and the recent therapeutic advances for neuromuscular disorders. [WATCH TIME: 3 minutes]
Managing New Therapies for Neuromuscular Diseases: Jinsy Andrews, MD, MSc
March 16th 2022The director of neuromuscular clinical trials at Columbia University Irving Medical Center shared her perspective on the challenges of familiarizing oneself with the influx of novel medications for neuromuscular diseases. [WATCH TIME: 3 minutes]
The Revolution in Limb-Girdle Muscular Dystrophy With Gene Therapies: Nicholas E. Johnson, MD, MSci
March 16th 2022The division chief of neuromuscular disorders and vice-chair of research at Virginia Commonwealth University offered his insight into the advances in genetic approaches to LGMD. [WATCH TIME: 2 minutes]
Diagnostic Delays and the Importance of the Physician-Patient Relationship: Amy Shinneman
March 15th 2022The 2022 MDA National Ambassador and patient with Bethlem myopathy spoke to the critical need to improve diagnosis delays in neuromuscular disorders and effective communication between patients and their physicians. [WATCH TIME: 2 minutes]
The Need to Elevate Patient Voices in Neuromuscular Disorders: Amy Shinneman
March 14th 2022The 2022 MDA National Ambassador and patient with Bethlem myopathy spoke to the importance of including the patient perspective at medical meetings to improve the care paradigm in neuromuscular disorders. [WATCH TIME: 3 minutes]
Nusinersen Shows Long-Term Safety, Benefits for Presymptomatic Infants With SMA
March 14th 2022Data from the phase 2 NURTURE study of nusinersen (Spinraza; Biogen) suggest that long-term treatment with the agent is beneficial, and point to the importance of newborn screening and early treatment for spinal muscular atrophy.
Characteristics of and Prevention Methods for Cerebral Palsy: Bhooma Aravamuthan, MD, DPhil
March 9th 2022The assistant professor of pediatric neurology at Washington University in St. Louis provided an overview of cerebral palsy and ways to reduce the risk of developing the condition. [WATCH TIME: 3 minutes]