Neuromuscular

Doug Kerr, MD, PhD, chief medical officer at Dyne Therapeutics, discusses the clinical development of DYNE-101 for myotonic dystrophy type 1 and DYNE-251 for Duchenne muscular dystrophy.

The FDA has approved Novartis’ intrathecal formulation of onasemnogene abeparvovec-brve, marketed as Itvisma, for patients ages 2 years and older living with spinal muscular atrophy.

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

Neurology News Network for the week ending November 22, 2025. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 21, 2025.

The senior director of research and real world data at Genome Medical talked about the benefits and challenges of genetic counselors adopting artificial intelligence tools. [WATCH TIME: 5 minutes]

Daniel Mikol, MD, PhD, vice president of neuroscience development at AbbVie, discussed how precision tools, AI, and patient partnership are reshaping the design and execution of modern neuroscience clinical trials.

The genetic counselor in the Department of Ophthalmology at the University of Pittsburgh Medical Center talked about the evolving role of genetic counselors in guiding patients through gene therapy. [WATCH TIME: 4 minutes]

KYV-101 shows promising efficacy in treating generalized myasthenia gravis, offering hope for durable remission through innovative CAR T-cell therapy.

Ryan Donaghy, MD, an assistant professor of neurology at Northwestern Medicine, discussed the evolving landscape of ALS clinical trials, including advances in biomarkers, genetics, and modern trial design.

Carrie Haverty, MS, CGC, NSGC president-elect, discusses the evolving role of genetic counselors, emphasizing personalized care, gene therapy advancements, and the need for multidisciplinary integration in health care.

Results of a new study funded by the ALS Association showed that the global prevalence of patients living with amyotrophic lateral sclerosis may increase by 25% by 2040.

The chief medical officer at Dyne Therapeutics highlighted z-rostudirsen’s ability to restore dystrophin and improve function across DMD severity levels, setting the stage for a potential 2026 accelerated approval. [WATCH TIME: 4 minutes]

Naji Gehchan, MD, MSc, chief medical and development officer Kyverna, discussed how KYV-101 may redefine generalized myasthenia gravis care through deep B-cell depletion and durable, drug-free remission.

Clene Nanomedicine prepares to submit an NDA for CNM-Au8, a promising ALS treatment, aiming for accelerated FDA approval by 2026.

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

Spencer Hutto, MD, associate program director of the adult neurology residency at Emory University, outlined the key diagnostic nuances, treatment considerations, and emerging therapeutic strategies shaping modern neurosarcoidosis care.

Neurology News Network for the week ending November 15, 2025. [WATCH TIME: 4 minutes]

The agency has required a postmarketing observational study to monitor safety for at least 12 months following gene therapy treatment in patients with Duchenne muscular dystrophy.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 14, 2025.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with James Berry, MD, MPH; and Jinsy Andrews, MD, MSc. [LISTEN TIME: 28 minutes]

The phase 3 FORTIFY trial of BBP-418 met its primary and key secondary interim analysis end points and demonstrated a safety profile consistent with prior studies in patients with LGMD2I/R9.

The executive vice president of public policy and advocacy at the Muscular Dystrophy Association discussed how collaboration can improve policy, funding, and care for patients with neuromuscular diseases. [WATCH TIME: 7 minutes]

The vice president and Asset Head of Rare and Ultra-Rare Diseases at UCB provided clinical perspectives on the FDA approval of Kygevvi, the first marketed treatment specific for patients with thymidine kinase 2 deficiency (Tk2d).

A genetic counselor-only clinic enhances access to genetic testing in pediatric neurology, proving efficient and effective for timely precision diagnostics.