At a NeurologyLive Clinical Forum in Philadelphia, Jessica D. Schulte, MD, PhD, of NYU Grossman School of Medicine, discussed how earlier recognition and systemic therapy options are changing the treatment paradigm for patients with NF1-associated plexiform neurofibromas.
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.
Adults with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive now have a second self-administration option for the complement inhibitor zilucoplan.
Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Nicholas J. Maragakis, MD; and Jinsy Andrews, MD, MSc, FAAN, FANA. [LISTEN TIME: 23 minutes]
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.
The designation follows phase 2 ClearMEMory data in which the oral NMDA receptor antagonist improved objective cognitive performance over placebo at 12 weeks.