Latest Conference Coverage

Two phase 3 ADAGIO trials test Cobenfy for Alzheimer’s agitation, assessing muscarinic modulation safety and efficacy as a potential new dementia care option.

The child neurologist at Children’s Hospital of Philadelphia discussed the real-world safety data of givinostat use in patients with Duchenne muscular dystrophy at the 2026 MDA Conference. [WATCH TIME: 5 minutes]

UK platform trial enrolls 1,600 with Parkinson’s to rapidly test telmisartan, terazosin and new candidates via shared placebo and virtual visits.

The professor of neurology at Cleveland Clinic’s Mellen Center for MS provided thoughts on how radiologic biomarkers may help shape the next generation of multiple sclerosis clinical trials. [WATCH TIME: 3 minutes]

Early BB-301 gene therapy data show durable swallowing gains and benign safety in OPMD dysphagia, hinting at disease modification.

Phase 3 data show Ultragenyx’s DTX301 gene therapy lowers ammonia in OTC deficiency, easing symptoms and reducing reliance on restrictive diets.

A phase 1/2 study aims to assess the safety and tolerability of nivudirsen, also known as BMN 351, in single ascending and multiple ascending doses among patients living with Duchenne.

A neurologist at Georgetown University, MedStar Health, discussed phase 2/3 data presented at the 2026 MDA Conference that supports efgartigimod as a novel treatment for inflammatory myopathies. [WATCH TIME: 2 minutes]

A retrospective cohort study reported that myocardial fibrosis detected by cardiac MRI was associated with higher fatigue scores among women carriers of Duchenne or Becker muscular dystrophy.

Neurology News Network for the week ending March 14, 2026. [WATCH TIME: 4 minutes]

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.

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.

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]