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, Laura Ann1 (AUTHOR) adangl@chop.edu, Sevagamoorthy, Anjana1 (AUTHOR), Sherbini, Omar1 (AUTHOR), Fraser, Jamie L.2,3 (AUTHOR), Bonkowsky, Joshua L.4,5 (AUTHOR), Gavazzi, Francesco1 (AUTHOR), D'Aiello, Russel1 (AUTHOR), Modesti, Nicholson B.1 (AUTHOR), Yu, Emily1 (AUTHOR), Mutua, Sylvia1 (AUTHOR), Kotes, Emma1 (AUTHOR), Shults, Justine6,7 (AUTHOR), Vincent, Ariel8 (AUTHOR), Emrick, Lisa T.9,10 (AUTHOR), Keller, Stephanie11 (AUTHOR), Van Haren, Keith P.12 (AUTHOR), Woidill, Sarah1 (AUTHOR), Barcelos, Isabella1 (AUTHOR), Pizzino, Amy1 (AUTHOR), Schmidt, Johanna L.1 (AUTHOR) |
| Source: | Molecular Genetics & Metabolism. May2024, Vol. 142 Issue 1, pN.PAG-N.PAG. 1p. |
| Database: | Academic Search Ultimate |
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