Efficacy of Intrathecal Adrabetadex in Infantile-Onset Niemann-Pick Disease Type C is Supported by Survival, Clinical, and Biomarker Data — Poster
Citation: Porter FD, et al. Efficacy of Intrathecal Adrabetadex in Infantile-Onset Niemann-Pick Disease Type C is Supported by Survival, Clinical, and Biomarker Data. Poster presented at: NORD Breakthrough Summit; October 2026.