Neuromuscular

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 chief medical officer at Dyne Therapeutics discussed the ACHIEVE trial’s early results, highlighting z-basivarsen’s potential to meaningfully improve muscle and CNS function in patients with myotonic dystrophy type 1. [WATCH TIME: 3 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).

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]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 7, 2025.

A new study presented at NSGC 2025 showed that patients living with amyotrophic lateral sclerosis considering medical aid in dying often experienced emotional and logistical challenges with the process.

A recent study highlights the link between genetic neurodevelopmental disorders and increased gastrointestinal issues in pediatric patients, emphasizing the need for targeted care.

Spingogenix presents promising data on SPG302, a daily tablet showing cognitive improvements in ALS patients with excellent safety profiles.

The chief medical officer at Dyne Therapeutics discussed the scientific rationale and clinical progress of z-basivarsen, an antisense oligonucleotide therapy designed to correct mis-splicing in myotonic dystrophy type 1. [WATCH TIME: 5 minutes]

Feedback from the FDA allows CervoMed to move forward with the proposed primary and secondary end point of the phase 3 trial assessing neflamapimod in dementia with Lewy bodies.

FDA issues a complete response letter for Biohaven's troriluzole, a potential treatment for spinocerebellar ataxia, citing data concerns.

The associate professor of physical medicine and rehabilitation at Virginia Commonwealth University discussed his passion for educating others about electrodiagnosis and ultrasound in neuromuscular disorders.