Extended Data Table 2 Design of the 39-week study of male and female NHP

From: Oral splicing modulator branaplam in Huntington’s disease: a phase 2 randomized controlled trial

  1. Animals were dosed twice weekly at a volume of 5 mL/kg. Animals were placed on a dosing holiday and were not dosed on Days 96, 99 and 103 (males) or Days 89, 92 and 96 (females). Group 1 received vehicle control article only.
  2. bUp to two animals/sex/group in groups 1 and 4 were designated for a 16-week (males; 112 days) or 17-week (females; 119 days) recovery phase (based on survival).
  3. cDue to dose-limiting toxicity, animals in groups 3 and 4 received the final dose on week 31 (for males) or week 30 (for females) instead of week 39; selected animals in Group 1 had an approximately equivalent shortened dosing regimen to provide controls for these groups.
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