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.
Feature|Articles|August 28, 2026
What’s Next in Spinal Muscular Atrophy: Pipeline Overview of Investigational Therapeutics
Author(s)Mariane Hernandez, Isabella Ciccone, MPH
In recognition of SMA Awareness Month, held annually throughout August, NeurologyLive® reviews the investigational agents currently moving through clinical development for spinal muscular atrophy.
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