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.

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.

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 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.

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

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.

After 90 days of treatment, investigators observed positive findings on microdystrophin expression, as well as reductions in markers of muscle injury and stress, and muscle breakdown and dystrophic regeneration.

Matthew B. Harms, MD, an associate professor of neurology at Columbia University Irving Medical Center, an MDA Care Center, gave clinical insights on the advances in ALS genetic research and what clinicians can expect from the ALS/FTD track at the 2025 MDA Clinical & Scientific Conference.

Neurology News Network. for the week ending February 16, 2025. [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 chronic demyelinating inflammatory polyneuropathy (CIDP).

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 14, 2024.

The clinical researcher at St. Jude Children's Research Hospital discussed the evolving treatment landscape for Charcot-Marie-Tooth disease, which will be featured in a session at the 2025 MDA Conference. [WATCH TIME: 7 minutes]

Expected to conclude in late 2026, the trial will feature 30 patients with Jordan’s syndrome, aged 9-45 years, who will be tested for a 24-week treatment period.

This new option, along with updated prescribing information, is expected to allow for easier administration and storage, enhancing the overall treatment experience.

A minor difference in relapse rates at month 8 between rituximab and placebo narrowed by month 12, indicating no long-term treatment advantage.

The kinase inhibitor was approved for patients with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

In a recent case report, a 46-year-old man living with chronic inflammatory demyelinating polyneuropathy experienced multiple relapses despite various conventional treatments; however, found promise in ofatumumab, an approved drug for multiple sclerosis.

Troriluzole, a new chemical entity and third-generation novel prodrug that modulates glutamate, would become the first approved agent for SCA, pending the FDA’s clearance.