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