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The professor of neurology at Columbia University Irving Medical Center highlighted the decades of research collaboration for patients with neuromuscular disease at the 2026 MDA conference.

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.

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.

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.