TSHA-102 Produces Multidomain Functional Gains in Rett Syndrome Matt Hoffman

Published Date: 17 Oct 2026

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.

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The designation follows phase 2 ClearMEMory data in which the oral NMDA receptor antagonist improved objective cognitive performance over placebo at 12 weeks.

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The Galassia trial will evaluate the safety and tolerability of intranasal cenegermin in about 60 children with spastic cerebral palsy across Italy.

New analyses found differences in functional outcomes over 2 years among patients aged 8 to 12 years treated with delandistrogene moxeparvovec, while separate findings characterized micro-dystrophin expression in children younger than 3 years.

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.

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.

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