Findings from the phase 3 TransportNPC study showed that Trappsol Cyclo missed the primary end point in Niemann-Pick type C, but subgroup and survival data support planned 2026 NDA.
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.
New data from a Cure SMA survey showed that children treated with disease-modifying therapy soon after birth walk more often and have fewer comorbidities than those treated later.
A new Bayesian network meta-analysis found nipocalimab's serious adverse event rates compared favorably with other gMG therapies, while overall adverse event rates were comparable.
An updated open-label extension analysis and new MRI data from the phase 3 EPIDYS trial, presented at the 2026 AANEM Annual Meeting, add to the evidence for givinostat's long-term effects on disease progression.