Neuromuscular

Tracey Dawson, PhD, senior vice president of U.S. Therapeutic Area Head of Neuroscience at Novartis, expanded on the recently launched SMAshing My Limits campaign, a community-driven project aimed to address needs of developing teens with SMA.

Serum NPTX2 emerges as a novel biomarker in ALS, with combined NPTX2 and NfL levels improving survival prediction and disease monitoring.

Regeneron's cemdisiran shows promising results in treating generalized myasthenia gravis, paving the way for a potential new drug application in 2026.

The FDA has accepted the IND for COYA 302, a Treg-targeting combination therapy for ALS, clearing the way for a pivotal phase 2 multicenter trial.

Efgartigimod shows promise in treating seronegative myasthenia gravis, offering hope for patients with limited options and paving the way for new therapies.

Here's some of what is coming soon to NeurologyLive® this week.

Phase 3 study demonstrates risdiplam's effectiveness in treating presymptomatic spinal muscular atrophy, showing improved outcomes for infants identified through newborn screening.

PTC Therapeutics faces FDA setback for vatiquinone, a potential Friedreich ataxia treatment, requiring further studies to demonstrate efficacy.

Explore the role of the complement system in autoimmune neuropathies like GBS and CIDP, and discover innovative therapeutic strategies targeting complement pathways.

Scholar Rock’s phase 3 SAPPHIRE trial of apitegromab revealed that the treatment led to improved motor function in patients with spinal muscular atrophy over a 52-week period.

Here's some of what is coming soon to NeurologyLive® this week.

Novartis revealed promising phase 3 trial results for ianalumab, a potential first treatment for Sjögren’s disease, showing significant disease activity reduction.

Catch up on any of the neurology news headlines you may have missed over the course of July 2025, compiled all into 1 place by the NeurologyLive® team.

Here's some of what is coming soon to NeurologyLive® this week.

Daniel Freedman, PhD, of MIT, gave clinical commentary on how Openwater’s Open-LIFU enables new research into conscious perception and potential clinical applications for conditions like pain and depression.

Vertex Pharmaceuticals halts VX-993 development for acute pain after phase 2 trial shows no significant efficacy compared with placebo.

Here's some of what is coming soon to NeurologyLive® this week.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 1, 2025.

Capricor Therapeutics navigates FDA challenges for deramiocel, aiming to advance treatment for DMD-related cardiomyopathy with promising trial data.

Roche faces regulatory challenges for Elevidys, an approved gene therapy for Duchenne muscular dystrophy, amid safety concerns and clinical trial setbacks.

Here's some of what is coming soon to NeurologyLive® this week.

A new phase 3 study will explore masitinib's potential as treatment for ALS, aiming to enhance patient outcomes and confirm earlier promising results.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 25, 2025.

AstraZeneca's gefurulimab shows promising results in treating generalized myasthenia gravis, offering patients a convenient, self-administered therapy option.

Neurology experts anticipate groundbreaking clinical trial results in 2025, potentially transforming treatment strategies for various neurological conditions.

Members from Nuvig Therapeutics discussed the rationale and therapeutic development of NVG-2089, a novel anti-inflammatory agent being studied for chronic inflammatory demyelinating polyneuropathy.

The FDA-cleared duo devices allow for a more simplified administration of the liquid medicine Hyqvia, reducing the number of steps needed to prepare for the infusion of 2 dual vial units or more.

Here's some of what is coming soon to NeurologyLive® this week.

The associate professor of neurology at Johns Hopkins detailed how artificial intelligence is shaping the future of neurology through its integration in clinical care, research, and medical education. [WATCH TIME: 4 minutes]

FDA halts Sarepta's Elevidys gene therapy distribution after three patient deaths, prioritizing safety amid ongoing clinical trial evaluations.