Neuromuscular

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.

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.

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.

Phase 1/2 long-term data showed tividenofusp alfa significantly reduced central and peripheral biomarkers to near-normal levels while improving liver volume, hearing thresholds, adaptive behavior, and cognition in most participants.

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 the International Stroke Conference (ISC).

The group of women neurologists provided commentary on the critical areas of women-focused research in neurology, from clinical trial participation to addressing workforce challenges.

Panelists explore the challenges and opportunities for women in neurology, offering practical advice for young professionals navigating the field.

In this episode, women neurologists share their personal experiences navigating challenges in neurology, offering insights into mentorship, work-life balance, and career advancement.

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

A group of empowering women leaders in neurology explore the transformative impact of women neurologists on the medical workforce and the strides toward a more inclusive neurology field.

UCB’s rozanolixizumab introduces two new self-administration methods, empowering patients with generalized myasthenia gravis (gMG) to manage their treatment with greater independence and control.

Deborah Benzil, MD, FAAS, vice chair of neurosurgery at Cleveland Clinic's Center for Spine Health, shared her extraordinary journey of resilience, from overcoming repeated rejections to becoming a trailblazer for women in neurosurgery, and reflects on the progress made and the challenges that remain.

A group of empowering women leaders in neurology explore the transformative impact of women neurologists on the medical workforce and the strides toward a more inclusive neurology field.

Neurology News Network. for the week ending February 2, 2025. [WATCH TIME: 4 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 Pompe disease.

The initial cohort of the EPISOD1 trial demonstrated no significant safety concerns for AMT-162, enabling progression to the next dose-escalation phase.

A new study identified key clinical and ultrasound markers that improved the differentiation between 2 conditions with overlapping features.

The chief medical advisor at the Muscular Dystrophy Association shared key highlights of what attendees can expect at the 2025 MDA Clinical and Scientific Conference. [WATCH TIME: 4 minutes]

Explore some of the most highly anticipated clinical trials with data readouts expected in the second half of 2025—key updates that researchers and clinicians in neurology won’t want to miss.

The submission was based on promising data from a phase 3 and phase 2 study, where treatment with apitegromab led to significant improvements in motor function among patients with SMA on SMN-targeted treatments.

The neurointensivist and assistant professor of neurology at Boston Medical Center gave insight on a 2024 paper establishing entrustable professional activities for neurocritical care advanced practice providers.

Early initiation of combined low‐dose rituximab therapy showed better improvements of chronic inflammatory demyelinating polyradiculoneuropathy in a short‐term than delayed initiation.