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Longitudinal Natural History Studies Based on Real-World Data in Rare Diseases: Opportunity and a Novel Approach

Laura Ann Adang,Anjana Sevagamoorthy,Omar Sherbini,Jamie L. Fraser,Joshua L. Bonkowsky,Francesco Gavazzi,Russel D'Aiello,Nicholson B. Modesti,Emily Yu,Sylvia Mutua, Emma Kotes,Justine Shults,Ariel Vincent,Lisa T. Emrick,Stephanie Keller,Keith P. Van Haren,Sarah Woidill,Isabella Barcelos,Amy Pizzino,Johanna L. Schmidt,Florian Eichler,Ali Fatemi,Adeline Vanderver

MOLECULAR GENETICS AND METABOLISM（2024）

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Key words
Rare disease,Natural history studies,Real world data,Novel approach,Longitudinal disease trajectory,Clinical trial readiness
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