Latest Conference Coverage

The chief medical officer at Edgewise discussed long-term MESA data showing sustained functional stabilization with sevasemten in Becker muscular dystrophy and its potential as a disease-modifying therapy.

Experts discussed new biomarker data from a phase 1/2 study, presented at the 2026 MDA Conference, of the gene therapy SGT-003 in boys living with Duchenne muscular dystrophy.

The 2026 MDA National Ambassador discussed the importance of connecting patients’ experiences with scientific understanding, as well as fostering a sense of community among those living with neuromuscular disease. [WATCH TIME: 2 minutes]

Phase 2 findings indicated that the investigational agent zervimesine exhibited a favorable safety profile and may slow disease progression in patients with mild to moderate dementia with Lewy bodies.

The assistant professor of medicine at the University of Toronto discussed population-level evidence suggesting MS may precede EBV infection in rare cases and its implications for disease pathogenesis and diagnosis.

A new retrospective analysis presented at AD/PD 2026 showed that cutaneous phosphorylated α-synuclein testing reduced diagnostic and management-related health care expenditures.

An analysis of baseline data from the phase 2b LUMA study suggested that keystroke dynamics captured through a smartphone-based typing task could provide a reliable method for assessing disease progression in Parkinson disease.

The research portfolio director at the Muscular Dystrophy Association highlighted the collaborative spirit, the expanding therapeutic pipeline, and the growing emphasis on patients’ voices in neuromuscular disease on display at MDA 2026. [WATCH TIME: 8 minutes]

Early ASPIRO trial data show autologous stem-cell dopamine precursors stay safe at 12 months, with improved PD motor scores and PET-confirmed grafting.

Phase 1 data suggest plasma GFAP levels may correlate with cognitive outcomes in patients with Alzheimer disease treated with troculeucel, an investigational autologous NK-cell therapy.

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.

At the 2026 MDA Conference, a neurologist at Nemours Children's Hospital discussed practical strategies for navigating insurance to ensure patients can access newly available neuromuscular therapies. [WATCH TIME: 3 minutes]

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.