Neuromuscular

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 28, 2025.

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.

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

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.

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.

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.

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.

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 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).

The vice president and Asset Head of Rare and Ultra-Rare Diseases at UCB discussed the landmark FDA approval of doxecitine/doxribitine, the first-ever therapy for thymidine kinase 2 deficiency (TK2d). [WATCH TIME: 3 minutes]

The vice president of Rare Disease Strategy at Worldwide Clinical Trials discussed how genetic counselors are becoming integral to the design, execution, and interpretation of clinical research. [WATCH TIME: 3 minutes]

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

A study reveals the intricate decision-making process caregivers face when considering gene therapy for Duchenne muscular dystrophy, emphasizing shared input and personal experiences.

Although their knowledge, experience, and access to genetic counselors varied, most licensed OB/GYNs across Ohio reported routinely offering carrier screening for spinal muscular atrophy.

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

Neurology News Network for the week ending November 8, 2025. [WATCH TIME: 4 minutes]