Neuromuscular

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]

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Elizabeth Head, PhD. [LISTEN TIME: 20 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.

Lewis B. Morgenstern, MD, reflects on his SEQUINS Hall of Fame recognition, the importance of health equity research, and his ongoing efforts to reduce stroke disparities.

Cheryl Bushnell, MD, MHS, a professor of neurology at Wake Forest University, discussed her SEQUINS Hall of Fame recognition, her work on sex differences in stroke risk, and her ongoing efforts to improve stroke care equity.

Administered intravenously in a single-dose vial, nipocalimab becomes the second FDA-approved therapy for common forms of generalized myasthenia gravis, and the first for younger patients living with the disease.

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

The chief scientific officer at Precision Biosciences provided clinical insights on the durable potential of ARCUS gene editing and the company’s next steps toward in-human trials for Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]

The neuromuscular neurologist at Nemours Children's Hospital in Florida talked about the increasing role of genomic medicine in neuromuscular disorders. [WATCH TIME: 4 minutes]

The chief scientific officer at Precision Biosciences provided clinical commentary on ARCUS gene editing platform and its potential application in Duchenne muscular dystrophy through a durable, AAV-delivered therapeutic strategy. [WATCH TIME: 4 minutes]

Results from the open-label extension of a phase 3 trial showed that treatment with ravulizumab sustained clinical benefits for up to 4 years in patients with generalized myasthenia gravis.

Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio, offered a clinical and translational overview of OPMD, highlighting current care challenges and the early promise of dual-action gene therapy strategies.