NEUROMUSCULAR

NeurologyLive's Neuromuscular Clinical Focus page focuses on the latest news and research for rare disorders like amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), and myasthenia gravis (MG).

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The Impact of the ENDEAR Trial Regimen

A discussion on the ENDEAR trial regimen and how an FDA approved treatment has impacted the management of spinal muscular atrophy.

Eculizumab Generates High Response Rates in Generalized Myasthenia Gravis

By the end of the open-label extension, 15-17% of patients showed clinically meaningful first response with eculizumab treatment.

Nusinersen Effective in Real-World SMA Treatment

Real-world, observational cohort data suggest that Biogen’s nusinersen (Spinraza) is safe and effective in the treatment of spinal muscular atrophy.

AT132 Gene Therapy Shows Early Phase Safety and Efficacy in X-Linked Myotubular Myopathy

Preliminary data from the ASPIRO study of Audentes Therapeutics resamirigene bilparvovec gene therapy suggests it is safe with improvements in neuromuscular and respiratory function.

Psychological Effects of SMA on Children and Adults

Dominic Pelle, MD: Improving Patient-Reported Outcomes in Spine Surgery

Centers of Excellence and Future Oral Medication

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About NeurologyLive's Neuromuscular Clinical Focus page:

The NeurologyLive Neuromuscular Clinical Focus page provides the latest clinical updates about neuromuscular diseases, with articles and videos covering the most up-to-date advancements in the field. Visitors to the clinical focus page will find information on emerging treatment options, as well as updates from the world’s top neurology conferences and annual meetings, including key coverage on rare disorders like ALS, DMD, and MD.

Additionally, articles and video editorial from neurologists and professionals specializing in neuromuscular conditions can be found on this page, offering expert guidance on current management strategies and potential neuromuscular therapy options.