News

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 13, 2026.

Phase 3 data show azetukalner cuts monthly focal seizures in refractory epilepsy, offering a novel KV7 option ahead of 2026 FDA filing.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with AAN President Natalia Rost, MD, MPH. [LISTEN TIME: 16 minutes]

Benjamin Greenberg, MD, internationally recognized NMOSD expert, discussed the growing use of recently approved therapies, the importance of early diagnosis, and investigational approaches, including biomarker development and CAR-T therapy, for NMOSD Awareness Month.

Interim data from the phase 1/2 AFFINITY DUCHENNE trial suggest RGX-202 gene therapy was well tolerated and produced robust microdystrophin expression with early functional improvements in boys with Duchenne muscular dystrophy.

A comparative analysis presented at the 2026 MDA Conference suggests the intrathecal gene therapy Itvisma may achieve similar motor outcomes to nusinersen and risdiplam in patients with spinal muscular atrophy.

Interim results from the phase 1/2 FORTIS trial suggest the investigational gene therapy AT845 was generally well tolerated and associated with stable respiratory and functional outcomes in adults with late-onset Pompe disease.

John Crowley, chief executive officer of the Biotechnology Innovation Organization, discusses the evolving biotechnology ecosystem, emerging therapeutic technologies, and ongoing policy challenges shaping the future of rare disease innovation.

SAFARI44 launches a phase 3 test of del-zota for DMD exon 44 skipping, tracking muscle function and dystrophin gains over 54 weeks.

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.

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 Seronegative generalized myasthenia gravis!

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]