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.
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March 21st 2023
Efgartigimod-treated patients consistently outperformed placebo regardless of concomitant medication, thymectomy status, and timing of diagnosis.
FDA to Hold AdComm Meeting on Investigational DMD Treatment SRP-9001
March 18th 2023The FDA has reversed course, deciding to hold an advisory committee meeting ahead of the May 29, 2023, action date for Sarepta’s investigational DMD agent SRP-9001 (also known as delandistrogene moxeparvovec).
Trofinetide Approval Brings Encouragement for Rett Disease, Rare Neurodevelopmental Disorders
March 14th 2023Jeffery Neul, MD, PhD, director of the Vanderbilt Kennedy Center, and professor of pediatrics at Vanderbilt University Medical Center, shared his reactions and thoughts to the approval of trofinetide.
NeurologyLive® Top Expert Interviews: Brain Awareness Week
March 14th 2023In honor of Brain Awareness Week, a group of experts in the care of patients with neurological conditions—Ronald C. Petersen, MD, PhD; Brian Grosberg, MD, FAHS; James Beck, PhD; Michael Levy, MD, PhD; Stanley H. Appel, MD —shared their perspectives on hot topics of treatment and management in neurology.
Health-Related Quality of Life Improvements Seen in Myasthenia Gravis With Efgartigimod
March 7th 2023Efgartigimod resulted in rapid impact on health-related quality of life, with improvements that were consistent across multiple measures and were similar for the effect seen across 2 treatment cycles.
An Overview of Therapeutic Options for Amyotrophic Lateral Sclerosis
March 4th 2023With an anticipated life expectancy of 3 years from the time of symptom onset, an effective treatment strategy is essential in ALS—and recent therapeutic progress has built a foundation of hope for the community.
Rare Disease Day: Expert Insights on Rare Neurological Diseases
March 1st 2023A group of experts in the care of patients with rare diseases—Emma Ciafaloni, MD, FAAN; Martina Bebin, MD, MDA; Timothy Miller, MD, PhD; George Small, MD; Bruce Cree, MD, PhD, MAS, FAAN—shared their perspectives on hot topics of treatment and management.
Rare Disease Day: Updates on Friedreich Ataxia and Myotonic Muscular Dystrophy
February 28th 2023In recognition of Rare Disease Day, the NeurologyLive® team offered an extensive update on the state of care and treatment for a few rare neurological diseases, including Pompe disease, Rett syndrome, among others.
Rare Disease Day: Updates on Rett Syndrome and Pompe Disease
February 28th 2023In recognition of Rare Disease Day, the NeurologyLive® team offered an extensive update on the state of care and treatment for a few rare neurological diseases, including Pompe disease, Rett syndrome, among others.
Clinician Perspectives on Black History Month: Jennifer Adrissi, MD
February 20th 2023To honor Black History Month, NeurologyLive® spoke with influential Black clinicians on the leaders they look up to, the ongoing fight to overcome racial disparities, and ways to encourage diversity in health care.