Extended Data Table 1 Demographic data for patients in natural history database

From: Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial

  1. Demographics of benchmark natural history data include genetic mutations, balanced male/female sex ratios, and other characteristics similar to the FIH participants.
  2. Abbreviations: ASPA, aspartoacylase gene; F, female; FIH, first-in-human participants; M, male; MSEL, Mullen Scales of Early Learning; NH, natural history patients; USA, United States of America.
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