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Advanced filters: Author: Arvind Kinhikar Clear advanced filters

  • Early results from the phase 2b VIBRANT-HD trial of the oral splicing modulator branaplam in Huntington’s disease suggested reduced cerebrospinal fluid huntingtin levels, but safety monitoring revealed signs of peripheral neurotoxicity, prompting early termination of the study.

    • Beth Borowsky
    • Harry Ramos
    • Blair R. Leavitt
    ResearchOpen Access
    Nature Medicine
    Volume: 32, P: 103-112