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Neuromuscular
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.
This Week on NeurologyLive — June 21, 2021
June 21, 2021
Article
Here's what is coming soon to NeurologyLive.
Efgartigimod as a Treatment for Generalized Myasthenia Gravis: James F. Howard, MD
June 20, 2021
Video
The Distinguished Professor of Neuromuscular Disease at UNC School of Medicine spoke to the results of the phase 3 ADAPT trial, and what else he’s like to see investigated with efgartigimod.
Blarcamesine Exceeds Enrollment, Higher Plasma Nusinersen Leads to Meaningful Improvements, Neurodevelopmental Outcomes Unchanged by Maternal ASMs
June 19, 2021
Video
Neurology News Network for the week ending June 19, 2021.
NeurologyLive Friday 5 — June 18, 2021
June 18, 2021
Article
Take 5 minutes to catch up on NeurologyLive's highlights from the week ending June 18, 2021.
ADAPT Trial of Efgartigimod and its Potential in gMG: James F. Howard, MD
June 16, 2021
Video
The Distinguished Professor of Neuromuscular Disease at UNC School of Medicine discussed the findings of the ADAPT trial and the promise efgartigimod has shown to be an effective agent for generalized myasthenia gravis.
Vamorolone Efficacious in Treatment of DMD, Superior to Prednisone in Safety
June 16, 2021
Article
Santhera and ReveraGen announced findings of the phase 2b VISION DMD study of the investigational therapy.
Myostatin Inhibition in Amyotrophic Lateral Sclerosis
June 16, 2021
Article
ALS has only recently been associated with causative or disease-modifying mutations in 20 genes that encode proteins with diverse functions, with promising therapeutic targets including proteins in pathways that regulate protein homeostasis.
Integrating Advanced Practice Clinicians Into Neurology: Calli L. Cook, NP, DNP
June 16, 2021
Video
The nurse practitioner at the Woodruff School of Nursing at Emory University detailed why a cultural shift is needed to incorporate APCs into the neurology space and thus improve multidisciplinary care.
Collaboration in ALS Research: Sabrina Paganoni, MD, PhD
June 15, 2021
Video
The investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital also discussed further research that needs to be done in ALS.
Higher Dose Nusinersen Shows Greater Potential for Clinical Benefit in SMA
June 15, 2021
Article
A pharmacokinetic/pharmacodynamic model indicated that the exposures predicted with a higher dose of nusinersen may lead to more clinically meaningful increases of at least 5 points on CHOP INTEND score.