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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.

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.

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.