News

The phase 1/2 VALOR trial is evaluating the safety and preliminary efficacy of the investigational gene therapy ASP2957 in infants with X-linked myotubular myopathy who require significant ventilatory support.

The agency has set a PDUFA date for August 22, 2026.

Long-term follow-up from an open-label phase 2 extension study suggests the investigational exon-skipping therapy brogidirsen was well tolerated over 4.5 years and may help maintain motor function in patients with Duchenne muscular dystrophy.

The FDA has approved leucovorin calcium (Wellcovorin) as the first treatment for patients with cerebral folate transport deficiency caused by variants in the FOLR1 gene.

The study design of the phase 3 STELLAR trials, presented at the 2026 MDA Conference, aims to investigate salanersen among presymptomatic infants with spinal muscular atrophy.

Interim phase 2 data from the FORWARD-53 study suggest the exon-skipping therapy WVE-N531 was well tolerated and produced sustained dystrophin expression, reduced fibrosis, and functional improvements in boys with DMD amenable to exon 53 skipping.

Phase 2 CANYON data show sevasemten keeps LVEF steady and NT-proBNP stable in adults with Becker muscular dystrophy, hinting at heart protection.

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

Polish real-world study shows children with SMA improve after switching from nusinersen or risdiplam to gene therapy, with expected side effects.

PrimeC shows significant survival gains and slowed progression in ALS phase 2b follow-up, fueling plans for larger late-stage trials.

Neurology News Network for the week ending March 7, 2026. [WATCH TIME: 5 minutes]

A recently published position statement from the American Headache Society recommends annual screening for migraine in girls and women, highlighting the potential role of tools like the ID Migraine screener to improve identification.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 6, 2026.

Large-scale proteomic and transcriptomic analyses position the synaptic protein NRN1 as a resilience-linked therapeutic candidate in Alzheimer disease, though validation remains preclinical.

Neuro-oncology experts examined how emerging cancer therapies may reshape the spectrum of paraneoplastic neurologic syndromes. [WATCH TIME: 4 minutes]

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Amaal Starling, MD, FAHS, FAAN. [LISTEN TIME: 16 minutes]

A Large cohort analysis of more than 159,000 adults found greater adherence to multiple healthy dietary patterns—particularly the DASH diet—was linked to a significantly lower risk of subjective cognitive decline in midlife.

The neurologist at LMU Munich University Hospital discussed newly published data supporting plasma brain-derived tau as a dynamic biomarker of infarct growth and treatment response in acute ischemic stroke. [WATCH TIME: 4 minutes]

Catch up on any of the neurology headlines you may have missed in February 2026, compiled into 1 place by the NeurologyLive® team.

A first-in-human SMA1 gene therapy trial doses infants with GB221 via intracisternal AAV delivery, aiming to boost motor outcomes while limiting toxicity.

A cross-sectional analysis of nearly 28,000 middle-aged adults found that excessive daytime sleepiness is strongly associated with both sleep characteristics and modifiable psychological factors, highlighting the need for integrated prevention strategies.

Pilot trial finds low-dose lithium is safe in mild cognitive impairment, hints at memory benefits, and guides larger studies for Alzheimer prevention.

A new study showed that higher baseline Epworth Sleepiness Scale scores were associated with greater early continuous positive airway pressure adherence in patients with obstructive sleep apnea.

Neuro-oncology experts discussed best practices for initiating immunomodulatory therapy in patients with PNS, factors influencing neurologic recovery, and the role of early specialty involvement and supportive care in optimizing long-term outcomes. [WATCH TIME: 4 minutes]

The associate professor of neurology at Mayo Clinic provided a breakdown of the real-world eptinezumab data from INFUSE and its implications for early, robust migraine intervention after CGRP failure.

Neal K. Shah, CEO of CareYaya Health Technologies, discusses how the largest workforce in the US maintains the safety of patients with dementia at home while remaining underpaid and unrecognized.

R. Scott Turner, PhD, MD, director of the memory disorders program at MedStar Georgetown University Hospital, discussed the importance of investigating environmental risk factors in Alzheimer disease.

Real-world INFUSE results show IV eptinezumab boosts good days and cuts monthly headache days for hard-to-treat migraine patients.