All 10 REVEAL Part A participants gained or regained at least 1 developmental milestone after a single intrathecal TSHA-102 dose, with no treatment-related SAEs reported.

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 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.

The professor of neurology and program director of Neuromuscular Medicine at UC Irvine, discusses phase 3 NIMBLE trial findings for cemdisiran in generalized myasthenia gravis, including its novel siRNA mechanism, efficacy, and hospitalization data.

New 48-week phase 3 data show many patients with generalized myasthenia gravis reached minimal symptom expression on telitacicept, and most who did sustained it.

Christen Kutz, PhD, PA-C, discusses a retrospective survey of neurology providers examining why patients with generalized myasthenia gravis are transitioning to nipocalimab and what real-world outcomes data suggest about its role in the treatment landscape.

PROPEL will test imeroprubart, an investigational FcRn blocker, against placebo in adults with mild to severe generalized myasthenia gravis.

New data from a Cure SMA survey showed that children treated with disease-modifying therapy soon after birth walk more often and have fewer comorbidities than those treated later.