Neuromuscular

A recent study shows that eteplirsen, a drug promoting dystrophin production, significantly slowed the decline in heart function, specifically left ventricular ejection fraction, in patients with Duchenne muscular dystrophy.

Spanning 14 weeks, the study evaluates age-appropriate dosing, safety, pharmacokinetics, and clinical effects through once-weekly injections of efgartigimod PH20 SC in juvenile myasthenia gravis.

Over the 5-year period, treatment-related adverse events with the gene therapy were mild or moderate, with most occurring within the first 90 days after infusion.

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 gene therapy for neuromuscular disorders.

Explore the key 2024 neurology guideline updates shaping clinical care across stroke, epilepsy, movement disorders, and more in this essential feature.

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

Neurology News Network. for the week ending March 8, 2025. [WATCH TIME: 3 minutes]

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

Based on the results of PROGRESS and the previous RELIEF-DPN-1 study, the 10 mg dose of pilavapadin was selected to move forward in clinical development for diabetic peripheral neuropathic pain.

With a scheduled PDUFA date of August 31, 2025, deramiocel looks to become the first marketed treatment for cardiomyopathy in DMD, one of the leading causes of death in this patient population.

Originally approved in 2007, eculizumab (Soliris; Alexion/AstraZeneca) is now available to treat both adult and pediatric patients with generalized myasthenia gravis, becoming the first available option for younger patients with the disease.

The pediatric neurologist at Children’s Hospital of Philadelphia gave perspective on some of the main issues the clinical community is figuring out with gene therapies and their integration to clinical practice. [WATCH TIME: 3 minutes]

Neurology News Network. for the week ending March 1, 2025. [WATCH TIME: 3 minutes]

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

Mary Schroth, MD, FAAP, FCCP, chief medical officer at Cure SMA, shed insight on the advances and promise for treating spinal muscular atrophy, in the context of Rare Disease Day.

The neurooncologist from the University of California Los Angeles provided clinical insight on the impact of a new treatment option for adults and children with neurofibromatosis type 1 that are not amenable to complete surgical resection. [WATCH TIME: 2 minutes]

The pediatric neurologist provided a clinical overview of the advances and roadblocks of gene therapy in neuromuscular disorders, which will be highlighted at the 2025 MDA Clinical & Scientific Conference.

Initial data reported from the FREEDOM-DM1 trial suggest PGN-EDODM1 has dose-dependent effects in patients with DM1, with further results from study cohorts expected in 2025 and 2026.

Entrada Therapeutics has recently received FDA clearance to move forward with a clinical trial for its investigational Duchenne muscular dystrophy therapy.

The associate professor of neurology at Columbia University provided commentary on the rapid advancements in translating genetic research into ALS treatments and the challenges that remain. [WATCH TIME: 5 minutes]

Neurology News Network. for the week ending February 23, 2025. [WATCH TIME: 3 minutes]

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

The pediatric neurologist at Children’s Hospital of Philadelphia provided background on a notable track session at the upcoming MDA Clinical & Scientific Conference covering updates in gene therapy for neuromuscular conditions. [WATCH TIME: 4 minutes]

A pair of lead neurologists at Baptist Health South Florida provided commentary on some of the promising developments expected to emerge in neurology over the coming months.

The FDA has assigned a PDUFA action date of August 19, 2025, and if approved, vatiquinone would be the first therapy for pediatric patients with Friedreich ataxia.