News

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 division chief of neuroimmunology at Brigham and Women’s Hospital discusses longitudinal EBNA-1 antibody data and its potential role as a complementary biomarker to distinguish MS from related neuroinflammatory diseases.

Since paraneoplastic neurologic syndromes remain underrecognized in oncology, experts recently discussed diagnostic challenges, treatment strategies, and the need for clinical vigilance.

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]

A global survey of neurologists and psychiatrists found that although tetrabenazine remains widely used for tardive dyskinesia and Huntington disease–related chorea, side effects frequently prevent optimal dose titration and lead to treatment discontinuation.

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.

New findings from a phase 3 trial conducted in China showed that solriamfetol boosted wakefulness and reduced daytime sleepiness in patients with obstructive sleep apnea, with manageable adverse effects.

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 post hoc analysis of the AMPA observational study found that adjunctive perampanel did not worsen daytime sleepiness over 12 months in patients with focal epilepsy while maintaining substantial seizure reduction.

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

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]

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is cerebroprotection in stroke!

In a retrospective cohort of 33 patients with chronic inflammatory demyelinating polyneuropathy, variants accounted for nearly two-thirds of cases, while intravenous immunoglobulin demonstrated high response rates across subtypes.

A randomized trial in patients with CIDP found that adding intravenous methylprednisolone to IVIg did not significantly increase remission rates and was associated with thromboembolic safety concerns.

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

Serum neurofilament light chain levels showed no significant treatment effect with rituximab compared with placebo, although exploratory trends suggested possible reductions in axonal injury among some treated patients.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 13, 2026.

Phase 3 data show azetukalner cuts monthly focal seizures in refractory epilepsy, offering a novel KV7 option ahead of 2026 FDA filing.

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.