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 associate vice president of clinical research at the Parkinson's Foundation discussed a large-scale replication of rs3115534-G, a noncoding GBA1 variant tied to Parkinson disease in people of African ancestry.
Leonard Dragone, MD, PhD, Disease Area Leader at Johnson & Johnson, discusses how nipocalimab is being incorporated into generalized MG care, what long-term data show, and where J&J sees the therapy’s role evolving.
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.