Oral splicing modulator branaplam in Huntington's disease: a phase 2 randomized controlled trial.

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Title: Oral splicing modulator branaplam in Huntington's disease: a phase 2 randomized controlled trial.
Authors: Borowsky B; Novartis Pharmaceuticals Corp., East Hanover, NJ, USA. beth.borowsky@novartis.com., Ramos H; Novartis Pharmaceuticals Corp., East Hanover, NJ, USA., Caputo A; Novartis Pharma AG, Basel, Switzerland., Hartmann A; Novartis Pharma AG, Basel, Switzerland., Faller T; Novartis Pharma AG, Basel, Switzerland., Peters T; Novartis Pharma AG, Basel, Switzerland., Sui Y; Novartis Pharmaceuticals Corp., East Hanover, NJ, USA., Liu F; Novartis Pharmaceuticals Corp., East Hanover, NJ, USA., Meadowcroft M; Novartis Biomedical Research Inc., Cambridge, MA, USA., David OJ; Novartis Pharma AG, Basel, Switzerland., Laisney M; Novartis Pharma AG, Basel, Switzerland., Kinhikar A; Novartis Biomedical Research Inc., Cambridge, MA, USA., Marder KS; Columbia University Irving Medical Center, New York, NY, USA., Tabrizi SJ; Queen Square Institute of Neurology, University College London, London, UK., Landwehrmeyer GB; University of Ulm, Ulm, Germany., Leavitt BR; Centre for Molecular Medicine and Therapeutics, BC Children's Hospital, University of British Columbia, Vancouver, British Columbia, Canada.
Source: Nature medicine [Nat Med] 2026 Jan; Vol. 32 (1), pp. 103-112. Date of Electronic Publication: 2026 Jan 05.
Publication Type: Journal Article; Clinical Trial, Phase II; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1546-170X
DOI:10.1038/s41591-025-04117-4