Adults with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive now have a second self-administration option for the complement inhibitor zilucoplan.
News|Articles|April 15, 2026
International Pompe Day 2026: Top News and Expert Insights
Author(s)Noah Stansfield
In observance of International Pompe Day, held annually on April 15, catch up on the past year’s news and expert insights related to this rare disease.
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According to the National Organization for Rare Disorders, Pompe disease occurs in approximately 1 in 40,000 births in the United States and the Netherlands; however, in some populations, the incidence is substantially higher.1 Over the past year, NeurologyLive® has covered news and clinical trial data updates related to the various therapies currently in development for Pompe disease and conducted interviews with experts to discern the potential for new research and treatments to affect the care paradigm.
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Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Nicholas J. Maragakis, MD; and Jinsy Andrews, MD, MSc, FAAN, FANA. [LISTEN TIME: 23 minutes]
Findings from the phase 3 METEOROID trial showed that satralizumab reduced the risk of relapse in patients with relapsing myelin oligodendrocyte glycoprotein antibody-associated disease.
The phase 1 ASCEND trial is testing a single intrathecal dose of the investigational AAV9 gene therapy INS1201 in young boys with Duchenne muscular dystrophy.
A quick-read roundup of 10 late-breaking abstracts from the 2026 MDS Congress, from phase 3 Parkinson disease readouts to long-term data in Huntington disease and Tourette syndrome, with the key results that matter for practice.
The designation follows phase 2 ClearMEMory data in which the oral NMDA receptor antagonist improved objective cognitive performance over placebo at 12 weeks.
An analysis of the phase 3 GEMZ trial presented at CNS 2026 showed that fenfluramine produced comparable seizure reductions and safety outcomes in children and adults with CDKL5 deficiency disorder.
Arimoclomol Plus Miglustat Shows Early, Sustained Slowing of Niemann-Pick Disease Type C Progression
A post hoc analysis of the phase 2/3 NPC002 trial presented at CNS 2026 showed that the benefit of arimoclomol plus miglustat in Niemann-Pick disease type C emerged as early as 3 months after treatment initiation.
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