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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
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  Data: Oral splicing modulator branaplam in Huntington's disease: a phase 2 randomized controlled trial.
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  Data: <searchLink fieldCode="AU" term="%22Borowsky+B%22">Borowsky B</searchLink>; Novartis Pharmaceuticals Corp., East Hanover, NJ, USA. beth.borowsky@novartis.com.<br /><searchLink fieldCode="AU" term="%22Ramos+H%22">Ramos H</searchLink>; Novartis Pharmaceuticals Corp., East Hanover, NJ, USA.<br /><searchLink fieldCode="AU" term="%22Caputo+A%22">Caputo A</searchLink>; Novartis Pharma AG, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Hartmann+A%22">Hartmann A</searchLink>; Novartis Pharma AG, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Faller+T%22">Faller T</searchLink>; Novartis Pharma AG, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Peters+T%22">Peters T</searchLink>; Novartis Pharma AG, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Sui+Y%22">Sui Y</searchLink>; Novartis Pharmaceuticals Corp., East Hanover, NJ, USA.<br /><searchLink fieldCode="AU" term="%22Liu+F%22">Liu F</searchLink>; Novartis Pharmaceuticals Corp., East Hanover, NJ, USA.<br /><searchLink fieldCode="AU" term="%22Meadowcroft+M%22">Meadowcroft M</searchLink>; Novartis Biomedical Research Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22David+OJ%22">David OJ</searchLink>; Novartis Pharma AG, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Laisney+M%22">Laisney M</searchLink>; Novartis Pharma AG, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Kinhikar+A%22">Kinhikar A</searchLink>; Novartis Biomedical Research Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Marder+KS%22">Marder KS</searchLink>; Columbia University Irving Medical Center, New York, NY, USA.<br /><searchLink fieldCode="AU" term="%22Tabrizi+SJ%22">Tabrizi SJ</searchLink>; Queen Square Institute of Neurology, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Landwehrmeyer+GB%22">Landwehrmeyer GB</searchLink>; University of Ulm, Ulm, Germany.<br /><searchLink fieldCode="AU" term="%22Leavitt+BR%22">Leavitt BR</searchLink>; Centre for Molecular Medicine and Therapeutics, BC Children's Hospital, University of British Columbia, Vancouver, British Columbia, Canada.
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  Data: <searchLink fieldCode="JN" term="%229502015%22">Nature medicine</searchLink> [Nat Med] 2026 Jan; Vol. 32 (1), pp. 103-112. <i>Date of Electronic Publication: </i>2026 Jan 05.
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