Neuromuscular

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.

The president and chief executive officer of the Biotechnology Innovation Organization shared insights on his keynote speech delivered at MDA 2026, reflecting on the evolving rare disease ecosystem. [WATCH TIME: 3 minutes]

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]

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

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.

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

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, also spoke about what he personally is most looking forward to at this year's meeting.

Global site reactivation for the MAGNITUDE-2 trial of nexiguran ziclumeran in hereditary transthyretin amyloidosis with polyneuropathy is underway, with enrollment completion expected in the second half of 2026.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending February 27, 2026.