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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 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.

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.