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Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach.

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Title: Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach.
Authors: Adang LA; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA. Electronic address: adangl@chop.edu., Sevagamoorthy A; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Sherbini O; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Fraser JL; Rare Disease Institute, Children's National Medical Center, Washington, DC, USA; Leukodystrophy and Myelin Disorders Program, Children's National Medical Center, Washington, DC, USA., Bonkowsky JL; Division of Pediatric Neurology, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, USA; Center for Personalized Medicine, Primary Children's Hospital, Salt Lake City, UT, USA., Gavazzi F; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., D'Aiello R; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Modesti NB; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Yu E; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Mutua S; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Kotes E; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Shults J; Department of Biomedical and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania, Philadelphia, PA, USA., Vincent A; CHOP Research Institute, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Emrick LT; Division of Neurology and Developmental Neuroscience in Department Pediatrics, Baylor College Medicine and Texas Children's Hospital, Houston, TX, USA; Department of Human and Molecular Genetics, Baylor College of Medicine, Houston, TX, USA., Keller S; Children's Healthcare of Atlanta Scottish Rite Hospital, Emory University School of Medicine, Atlanta, GA, USA., Van Haren KP; Department of Neurology, Stanford University, Stanford, CA, USA., Woidill S; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Barcelos I; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Pizzino A; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Schmidt JL; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Eichler F; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA., Fatemi A; Moser Center for Leukodystrophies, Kennedy Krieger Institute, Baltimore, MD, USA; Departments of Neurology & Pediatrics, Johns Hopkins School of Medicine, Baltimore, MD, USA., Vanderver A; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Source: Molecular genetics and metabolism [Mol Genet Metab] 2024 May; Vol. 142 (1), pp. 108453. Date of Electronic Publication: 2024 Mar 18.
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Review
Journal Info: Publisher: Academic Press Country of Publication: United States NLM ID: 9805456 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1096-7206 (Electronic) Linking ISSN: 10967192 NLM ISO Abbreviation: Mol Genet Metab Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach.
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  Data: <searchLink fieldCode="AU" term="%22Adang+LA%22">Adang LA</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA. Electronic address: adangl@chop.edu.<br /><searchLink fieldCode="AU" term="%22Sevagamoorthy+A%22">Sevagamoorthy A</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Sherbini+O%22">Sherbini O</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Fraser+JL%22">Fraser JL</searchLink>; Rare Disease Institute, Children's National Medical Center, Washington, DC, USA; Leukodystrophy and Myelin Disorders Program, Children's National Medical Center, Washington, DC, USA.<br /><searchLink fieldCode="AU" term="%22Bonkowsky+JL%22">Bonkowsky JL</searchLink>; Division of Pediatric Neurology, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, USA; Center for Personalized Medicine, Primary Children's Hospital, Salt Lake City, UT, USA.<br /><searchLink fieldCode="AU" term="%22Gavazzi+F%22">Gavazzi F</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22D'Aiello+R%22">D'Aiello R</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Modesti+NB%22">Modesti NB</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Yu+E%22">Yu E</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Mutua+S%22">Mutua S</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Kotes+E%22">Kotes E</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Shults+J%22">Shults J</searchLink>; Department of Biomedical and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Vincent+A%22">Vincent A</searchLink>; CHOP Research Institute, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Emrick+LT%22">Emrick LT</searchLink>; Division of Neurology and Developmental Neuroscience in Department Pediatrics, Baylor College Medicine and Texas Children's Hospital, Houston, TX, USA; Department of Human and Molecular Genetics, Baylor College of Medicine, Houston, TX, USA.<br /><searchLink fieldCode="AU" term="%22Keller+S%22">Keller S</searchLink>; Children's Healthcare of Atlanta Scottish Rite Hospital, Emory University School of Medicine, Atlanta, GA, USA.<br /><searchLink fieldCode="AU" term="%22Van+Haren+KP%22">Van Haren KP</searchLink>; Department of Neurology, Stanford University, Stanford, CA, USA.<br /><searchLink fieldCode="AU" term="%22Woidill+S%22">Woidill S</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Barcelos+I%22">Barcelos I</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Pizzino+A%22">Pizzino A</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Schmidt+JL%22">Schmidt JL</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Eichler+F%22">Eichler F</searchLink>; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Fatemi+A%22">Fatemi A</searchLink>; Moser Center for Leukodystrophies, Kennedy Krieger Institute, Baltimore, MD, USA; Departments of Neurology & Pediatrics, Johns Hopkins School of Medicine, Baltimore, MD, USA.<br /><searchLink fieldCode="AU" term="%22Vanderver+A%22">Vanderver A</searchLink>; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
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  Data: <searchLink fieldCode="JN" term="%229805456%22">Molecular genetics and metabolism</searchLink> [Mol Genet Metab] 2024 May; Vol. 142 (1), pp. 108453. <i>Date of Electronic Publication: </i>2024 Mar 18.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Academic+Press%22">Academic Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>9805456 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1096-7206 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2210967192%22">10967192 </searchLink><i>NLM ISO Abbreviation: </i>Mol Genet Metab <i>Subsets: </i>MEDLINE
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