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. |
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| 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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