How brain processes are rewired in Parkinson's disease

Published Date: 02 Mar 2023

Researchers from the University of Queensland have confirmed that genes associated with the risk of Parkinson's disease also contribute to the production of cellular debris in the brain.

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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 phase 1 ASCEND trial is testing a single intrathecal dose of the investigational AAV9 gene therapy INS1201 in young boys with Duchenne muscular dystrophy.

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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.

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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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