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.
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.