LRRK2-targeting antisense oligonucleotide in Parkinson's disease: a phase 1 randomized controlled trial.

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Title: LRRK2-targeting antisense oligonucleotide in Parkinson's disease: a phase 1 randomized controlled trial.
Authors: Mabrouk OS; Biogen Inc, Cambridge, MA, USA. omar.mabrouk@biogen.com., Tichler B; Biogen International GmbH, Baar, Switzerland., Arnold HM; Biogen Inc, Cambridge, MA, USA., Thoma EC; Biogen International GmbH, Baar, Switzerland., Alexanian SM; Biogen Inc, Cambridge, MA, USA., Chen J; Biogen Inc, Cambridge, MA, USA., Liu TY; Biogen Inc, Cambridge, MA, USA., Hirschhorn B; Biogen Inc, Cambridge, MA, USA., Llorens Arenas R; Biogen Inc, Cambridge, MA, USA., Annand JW; Biogen Inc, Cambridge, MA, USA., Kapadnis U; Biogen Inc, Cambridge, MA, USA., Shomo AA; Biogen Inc, Cambridge, MA, USA., Glajch KE; Biogen Inc, Cambridge, MA, USA.; Takeda, Cambridge, MA, USA., Ferber K; Biogen Inc, Cambridge, MA, USA., Moroishi Y; Biogen Inc, Cambridge, MA, USA., Czerkowicz J; Biogen Inc, Cambridge, MA, USA., Inra J; Biogen Inc, Cambridge, MA, USA., Postuma RB; Department of Neurology, McGill University, Montreal Neurological Institute, Montreal, Quebec, Canada., Gurevich T; Movement Disorders Unit, Neurological Institute, Tel Aviv Sourasky Medical Center, Tel Aviv University, Tel Aviv-Yafo, Israel., Mir P; Unidad de Trastornos del Movimiento, Servicio de Neurología, Instituto de Biomedicina de Sevilla, IBiS/Hospital Universitario Virgen del Rocío/CSIC/Universidad de Sevilla, Seville, Spain.; Centro de Investigación Biomédica en Red sobre Enfermedades Neurodegenerativas, Instituto de Salud Carlos III, Madrid, Spain.; Departamento de Medicina, Facultad de Medicina, Universidad de Sevilla, Seville, Spain., Morris HR; Department of Clinical and Movement Neuroscience, UCL Queen Square Institute of Neurology, London, UK., Aldred J; Selkirk Neurology & Inland Northwest Research, Spokane, WA, USA., Brodsky MA; Oregon Health & Science University, Portland, OR, USA., Ellenbogen A; Michigan Institute for Neurological Disorders, Farmington Hills, MI, USA., Larson D; Parkinson's Disease & Movement Disorders Center, Department of Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL, USA., Tolleson CM; The University of Tennessee Medical Center, Knoxville, TN, USA., Siderowf A; University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA., Tzoulis C; Neuro-SysMed, Department of Neurology, Haukeland University Hospital, Bergen, Norway.; Department of Clinical Medicine, University of Bergen, Bergen, Norway.; K.G. Jebsen Center for Translational Research in Parkinson's Disease, University of Bergen, Bergen, Norway., Balaguer E; Hospital Universitari General de Catalunya, Barcelona, Spain., Marti MJ; Unidad de Parkinson y Trastornos del Movimiento, Hospital Clínic de Barcelona, IDIBAPS, CIBER-ISCIII, Barcelona, Spain., Zhao HT; Ionis, Carlsbad, CA, USA., Kordasiewicz HB; Ionis, Carlsbad, CA, USA., Lane R; Ionis, Carlsbad, CA, USA., Hirst WD; Biogen Inc, Cambridge, MA, USA.; DaCapo Brainscience, Cambridge, MA, USA., Fradette S; Biogen Inc, Cambridge, MA, USA., Graham DL; Biogen Inc, Cambridge, MA, USA.
Source: Nature medicine [Nat Med] 2026 Apr; Vol. 32 (4), pp. 1421-1431. Date of Electronic Publication: 2026 Mar 24.
Publication Type: Journal Article; Randomized Controlled Trial; Clinical Trial, Phase I
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/s41591-026-04262-4