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.
Pivotal AFFINITY DUCHENNE results showed microdystrophin expression above the prespecified threshold in 93% of evaluable participants, with preliminary functional findings favoring treatment over external controls.
Three-year extension data from the phase 3 IB1001-301 trial show levacetylleucine slowed disease progression in both adults and children with Niemann-Pick disease type C.
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 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.
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.