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.

Episode 37: The Impact of Awareness Efforts in ALS

"Mind Moments," a podcast from NeurologyLive, brings you an exclusive interview with Matthew B. Harms, MD.

Case-Based Insights: Expert Perspectives on the Treatment of Adult-Onset SMA

Expert neurologists discuss patient cases involving the treatment of adult-onset spinal muscular atrophy (SMA).

NeuroVoices: Yung Chyung, MD, on the Advantages of Apitegromab in Early SMA Treatment

The chief medical officer of Scholar Rock described how the investigational apitegromab aims to fill some of the current unmet needs for patients with spinal muscular atrophy.

Risdiplam Gets Priority Review for Presymptomatic SMA in Babies Under 2 Months

January 25, 2022

Article

The submission to the FDA is supported by interim data from the RAINBOWFISH trial (NCT03779334), as all 5 babies treated with risdiplam maintained the ability to swallow and feed exclusively orally after 12 months.

Application Accepted for Oral Edaravone in Amyotrophic Lateral Sclerosis

January 24, 2022

Article

The FDA accepted the new drug application for priority review, setting a PDUFA action date of May 12, 2022.

This Week on NeurologyLive® — January 24, 2022

January 24, 2022

Article

Here's what is coming soon to NeurologyLive®.

YTX-7739 Trials Suspended, ENSURE-2 Study of IMU-838 Initiated, Behavioral Involvement of FTD in ALS

January 22, 2022

Video

Neurology News Network for the week ending January 22, 2022. [WATCH TIME: 4 minutes]

NeurologyLive® Friday 5 — January 21, 2022

January 21, 2022

Article

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending January 21, 2022.

Compassionate Use Clinical Trial of FGF-1 Initiated for Amyotrophic Lateral Sclerosis

January 19, 2022

Article

Zhittya Genesis Medicine announced the trial to evaluate its biological drug, FGF-1, which is administered via an intranasal delivery device, in a cohort of individuals with mild to moderate ALS.

Cognitive and Behavioral Involvement in ALS May Be Distinct Phenotype

January 17, 2022

Article

Those who had crossovers of ALS and frontotemporal dementia had structural and functional resemblance to the patterns of damage of those with only behavioral variant frontotemporal dementia.

This Week on NeurologyLive® — January 17, 2022

January 17, 2022

Article

Here's what is coming soon to NeurologyLive®.

NeurologyLive® Top Expert Interviews: December 2021

January 14, 2022

Article

Expert clinicians offer their insight on costs of Alzheimer drug development, a new agent for generalized myasthenia gravis, migraine in the emergency department, educational sleep medicine “boot camps”, AES 2021, and more.

NeurologyLive® Friday 5 — January 14, 2022

January 14, 2022

Article

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending January 14, 2022.