Latest Conference Coverage

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.

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]

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.

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.

A clinician and researcher at the University of California, San Francisco highlighted how agentic AI frameworks are being integrated into real-world multiple sclerosis research to streamline big data analysis and accelerate clinically meaningful discovery. [Watch Time: 5 minutes]

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.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Lauren Sansing, MD, MS, FAHA, FANA. [LISTEN TIME: 27 minutes]

The senior research investigator at the New York Stem Cell Foundation answered questions about the research efforts needed to advance iPSC human models toward clinical relevance for patients with multiple sclerosis. [WATCH TIME: 4 minutes]

Off-label rimegepant was associated with patient-reported acute migraine improvement and no new safety signals in a small retrospective review of adolescent patients.

The head of the Phase I Clinical Research Unit at Beijing Tiantan Hospital discussed positive clinical trial findings for a dual-target neuroprotectant in patients with acute ischemic stroke presented at ISC 2026. [WATCH TIME: 3 minutes]

The professor of medicine at McMaster University discussed findings from OCEANIC-STROKE, where treatment with asundexian reduced the risk of recurrent stroke without increasing major hemorrhage in patients. [WATCH TIME: 4 minutes]

Experts shared their clinical perspectives on trending topics at the 2026 Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum, held February 5-7, 2026.

An associate professor in neurosurgery at Duke University discussed his presentation on novel meningeal immune interactions and interferon signaling in multiple sclerosis at the ACTRIMS Forum 2026. [WATCH TIME: 3 minutes]

Phase 2 trials suggest LT3001 is safe in acute ischemic stroke and may boost day-90 recovery, paving the way for a global Phase 3.

Leaders from Brainomix provided commentary on data from ISC 2026, discussing how automated net water uptake from routine CT imaging may refine stroke severity assessment, thrombectomy selection, and system-level treatment equity.

The chief medical advisor at the Muscular Dystrophy Association also discussed what he personally is looking forward to at the conference this year. [WATCH TIME: 4 minutes]

A pediatric neurologist at the UCL Institute of Neurology in London, England discussed how pediatric-onset MS is marked by intense inflammation, unique neurodevelopmental considerations, and evolving treatment paradigms at ACTRIMS 2026. [WATCH TIME: 5 minutes]

High-dose constraint-induced therapy boosts arm function in toddlers after perinatal stroke, with gains lasting 6 months in phase 3 trial.

The professor of neurology at Cleveland Clinic gave insights on hematopoietic stem cell transplantation, CAR T-cell strategies, and the ongoing challenges of developing immune reconstitution and repair therapies in multiple sclerosis.

Andrew Russman, DO, director of Cleveland Clinic’s Comprehensive Stroke Center, provided an analysis of the phase 3 OCEANIC-STROKE data of asundexian in patients with noncardioembolic ischemic stroke or high-risk transient ischemic attack.

A professor of neurology from the University of Barcelona discussed the clinical implications of adding intra-arterial alteplase after thrombectomy in patients with Large-vessel occlusion (LVO) acute ischemic stroke at ISC 2026.