Neuromuscular

The research portfolio director at the Muscular Dystrophy Association discussed the evolving ALS therapeutic landscape, highlighting advances in genetic and RNA-based interventions. [WATCH TIME: 6 minutes]

Seif Elbualy, MD, discusses his role at the Brain and Spine Symposium and shares insights on the future of interventional pain management, evolving techniques, and pharmaceutical innovation.

Priya Kishnani, MD, division chief of Medical Genetics at Duke University, reflects on Pompe disease’s historical and scientific evolution, from early enzyme therapy to next-generation treatment strategies and newborn screening breakthroughs.

New data from the HELIOS trial shows AMX0035 improves glycemic control and stabilizes symptoms in Wolfram syndrome, paving the way for future studies.

Committee chairs Vincent Timmerman, PhD, MSc, and Kathrin Doppler, MD, provide a preview of the 2025 PNS Annual Meeting, giving clinicians an inside look at what to expect from the upcoming conference.

The chief executive officer and founder at CureDuchenne shared her emotional journey from her son’s diagnosis of Duchenne to embracing a new path focused on advocacy, adaptation, and resilience. [WATCH TIME: 4 minutes]

Phase 1b data show oral microbiome therapy MaaT033 slows ALS progression and is well tolerated, supporting future phase 2 development efforts.

This closing episode looks ahead to the future of SMA care, examining how updated best practices aim to accelerate access, optimize outcomes, and fuel ongoing progress.

Here's some of what is coming soon to NeurologyLive® this week.

This episode tackles how proactive, interdisciplinary care and dedicated coordination models are transforming the delivery of SMA treatment across clinical settings.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on B-cell depleting medications for MS.

Panelists comment on how treatment decisions for adolescents and adults with SMA are shaped by lifestyle, quality of life, logistical realities, and access to care.

Neurology News Network. for the week ending May 10, 2025. [WATCH TIME: 4 minutes]

Catch up on any of the neurology news headlines you may have missed over the course of April 2025, compiled all into one place by the NeurologyLive® team.

A study involving 20 patients with ALS found that longitudinal muscle MRI detected progressive atrophy and correlated with muscle strength loss, supporting its potential as a biomarker for ALS progression and clinical trials.

This episode explores treatment considerations for patients already living with SMA, emphasizing individualized goals, medical complexity, and the value of subtle clinical improvements.

In this episode, neurologists unpack the urgent and complex treatment decisions in newly diagnosed SMA, highlighting clinical factors, family input, and the importance of shared decision-making.

The research portfolio director at the Muscular Dystrophy Association highlighted advances in gene-targeted therapies and biomarker development as potential key drivers of progress in ALS.

Sunny Brous, a patient advocate living with ALS, shared how her decade-long journey with the disease shaped her mission to build connection, advocate for change, and empower the ALS community.

In episode 1, panelists explore the evolving SMA treatment landscape, highlighting updated best practices shaped by new therapies, newborn screening, and international expert consensus.

The professor of neurology at the University of Buffalo talked about the clinical promise of nipocalimab and its role in broadening treatment options for patients with myasthenia gravis, including adolescents. [WATCH TIME: 4 minutes]

Capricor Therapeutics reported that it remains on track for PDUFA target action date of August 31, 2025 and noted that advisory committee meeting is to be held in advance.

The expanded access program enables the collection of real-world data on the safety and efficacy of SPG302, which may support its clinical development for the treatment of amyotrophic lateral sclerosis.

Here's some of what is coming soon to NeurologyLive® this week.

The research portfolio director at the Muscular Dystrophy Association talked about recent advances in ALS research highlight the promise of gene-targeted treatments and the increasing utility of biomarkers for diagnosis. [WATCH TIME: 3 minutes]

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on the Peripheral Nerve Society annual meeting.

Neurology News Network. for the week ending May 3, 2025. [WATCH TIME: 3 minutes]

Moira Kapral, MD, MSc, reflects on her SEQUINS Hall of Fame recognition, the evolution of brain health equity, and her work addressing disparities in stroke care.

Robert J. Adams, MS, MD, reflects on his SEQUINS Hall of Fame recognition, his work on stroke prevention, and the importance of equity in brain health.

Barbara Vickrey, MD, MPH, discusses her SEQUINS Hall of Fame honor, the evolution of healthcare delivery models, and the role of mentorship in fostering equity in neurology.