Neuromuscular

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).

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.

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.

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.

The U.S. HHS announced 15 drugs, including deutetrabenazine, will undergo Medicare Part D price negotiations to lower costs for seniors by 2027.

Previously, Argenx's efgartigimod alfa and hyaluronidase-qvfc was granted orphan drug designation for the treatment of chronic inflammatory demyelinating polyneuropathy in Japan.

Crossover-treated patients demonstrated clinically meaningful and statistically significant improvements in functional outcomes despite being older at the time of treatment.

Neurology News Network. for the week ending January 25, 2025. [WATCH TIME: 4 minutes]

In the phase 2/3 DEVOTE study, a higher dose of nusinersen met its primary end point at 6 months, achieving a statistically significant improvement in motor function among treatment-naïve symptomatic infants with SMA.

Take a look at some of the most-anticipated FDA pending approvals expected in 2025 that researchers and clinicians in neurology should keep an eye out on.

The study highlighted tofersen's potential to slow ALS progression in patients with SOD1 mutations, showing reduced neurofilament levels, functional stabilization, and improved muscle strength, despite mild to moderate adverse events.

Robert Rothrock, MD, a spinal neurosurgeon at Baptist Health Miami Neuroscience Institute, provided commentary on key topics in spine health amid the 2025 Baptist Health Spine Symposium.

The professor and chair of the Department of Molecular Pathobiology at NYU College of Dentistry discussed how a recently published preclinical study adds to the understanding of neuropilin-1 in chronic pain. [WATCH TIME: 3 minutes]

A new study highlighted the importance of initiating immunotherapy promptly in chronic inflammatory demyelinating polyneuropathy to prevent axonal damage and disability progression.

A study highlighted corneal confocal microscopy as a promising noninvasive tool for tracking sensory nerve damage in chronic inflammatory demyelinating polyradiculoneuropathy.

Nipocalimab’s biologics license application is supported by the phase 3 Vivacity-MG3 trial, in which the agent demonstrated superiority over placebo plus standard-of-care over a 24-week period.

Early treatment of CIDP, within 1-year of onset, is associated with better long-term outcomes, highlighting key prognostic factors and treatment timing.

The professor and chair of the Department of Molecular Pathobiology at NYU College of Dentistry discussed groundbreaking research on chronic pain, focusing on the discovery of new mechanisms involving nerve growth factor and its receptors.