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Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study.

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第一作者: Leonard H,van den Berg
第一单位: UMC Utrecht Brain Center, Department of Neurology, UMC Utrecht, Utrecht, Netherlands. Electronic address: l.h.vandenBerg@umcutrecht.nl.
作者单位: UMC Utrecht Brain Center, Department of Neurology, UMC Utrecht, Utrecht, Netherlands. Electronic address: l.h.vandenBerg@umcutrecht.nl. [1] Department of Neurology, Johns Hopkins University, Baltimore, MD, USA. [2] Sheffield Institute for Translational Neuroscience, University of Sheffield, Sheffield, UK; National Institute for Health and Care Research Sheffield Biomedical Research Centre and Clinical Research Facility, University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK. [3] Sean M Healey and AMG Center for ALS, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. [4] Department of Neurology, University of Miami Miller School of Medicine, Miami, FL, USA. [5] Department of Neurology, Washington University School of Medicine in St Louis, St Louis, MO, USA. [6] Montreal Neurological Institute and Hospital, Montreal, Canada. [7] Department of Neurology, Emory University, Atlanta, GA, USA. [8] School of Medicine, Trinity College, Dublin, Ireland. [9] University College London Institute of Neurology and National Institute for Health and Care Research (NIHR) University College London Hospitals (UCLH) Clinical Research Facility and Biomedical Research Centre, London, UK. [10] Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada. [11] Department of Neurology, Mayo Clinic, Jacksonville, FL, USA. [12] Bryan Physicians Network, Lincoln, NE, USA; Department of Neurological Sciences, University of Nebraska Medical Center, Omaha, NE, USA. [13] Department of Neurosciences, University of California San Diego Health, San Diego, CA, USA. [14] UK Dementia Research Institute, King's College London, London, UK. [15] Neuromuscular Diseases Unit/ALS Clinic, Kantonsspital St Gallen, Switzerland. [16] Division of Neurology, Department of Medicine, Sunnybrook Health Sciences Centre, University of Toronto, Toronto, Canada. [17] Ionis Pharmaceuticals, Carlsbad, CA, USA. [18] Biogen, Cambridge, MA, USA. [19]
DOI 10.1016/S1474-4422(24)00216-3
PMID 39059407
发布时间 2025-01-30
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