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Trials for Slowly Progressive Neurogenetic Diseases Need Surrogate Endpoints.

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Title: Trials for Slowly Progressive Neurogenetic Diseases Need Surrogate Endpoints.
Authors: Reilly MM; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK., Herrmann DN; Department of Neurology, University of Rochester, Rochester, NY., Pareyson D; Unit of Rare Neurodegenerative and Neurometabolic Diseases, Department of Clinical Neurosciences, Fondazione IRRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Scherer SS; Department of Neurology, The Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA., Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN., Züchner S; Dr. John T. Macdonald Foundation Department of Human Genetics and John P. Hussman Institute for Human Genomics, University of Miami Miller School of Medicine, Miami, FL., Burns J; Sydney School of Health Sciences, University of Sydney, Sydney, Australia., Shy ME; Department of Neurology, Carver College of Medicine, University of Iowa, Iowa, IA.
Source: Annals of neurology [Ann Neurol] 2023 May; Vol. 93 (5), pp. 906-910. Date of Electronic Publication: 2023 Mar 21.
Publication Type: Journal Article; Review; Research Support, Non-U.S. Gov't; Research Support, N.I.H., Extramural
Journal Info: Publisher: Wiley-Liss Country of Publication: United States NLM ID: 7707449 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1531-8249 (Electronic) Linking ISSN: 03645134 NLM ISO Abbreviation: Ann Neurol Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Trials for Slowly Progressive Neurogenetic Diseases Need Surrogate Endpoints.
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  Data: <searchLink fieldCode="AU" term="%22Reilly+MM%22">Reilly MM</searchLink>; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.<br /><searchLink fieldCode="AU" term="%22Herrmann+DN%22">Herrmann DN</searchLink>; Department of Neurology, University of Rochester, Rochester, NY.<br /><searchLink fieldCode="AU" term="%22Pareyson+D%22">Pareyson D</searchLink>; Unit of Rare Neurodegenerative and Neurometabolic Diseases, Department of Clinical Neurosciences, Fondazione IRRCCS Istituto Neurologico Carlo Besta, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Scherer+SS%22">Scherer SS</searchLink>; Department of Neurology, The Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.<br /><searchLink fieldCode="AU" term="%22Finkel+RS%22">Finkel RS</searchLink>; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN.<br /><searchLink fieldCode="AU" term="%22Züchner+S%22">Züchner S</searchLink>; Dr. John T. Macdonald Foundation Department of Human Genetics and John P. Hussman Institute for Human Genomics, University of Miami Miller School of Medicine, Miami, FL.<br /><searchLink fieldCode="AU" term="%22Burns+J%22">Burns J</searchLink>; Sydney School of Health Sciences, University of Sydney, Sydney, Australia.<br /><searchLink fieldCode="AU" term="%22Shy+ME%22">Shy ME</searchLink>; Department of Neurology, Carver College of Medicine, University of Iowa, Iowa, IA.
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  Data: <searchLink fieldCode="JN" term="%227707449%22">Annals of neurology</searchLink> [Ann Neurol] 2023 May; Vol. 93 (5), pp. 906-910. <i>Date of Electronic Publication: </i>2023 Mar 21.
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              Text: 2023 May
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