Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach. - Adang, Laura Ann; Sevagamoorthy, Anjana; Sherbini, Omar; Fraser, Jamie L.; Bonkowsky, Joshua L.; Gavazzi, Francesco; D'Aiello, Russel; Modesti, Nicholson B.; Yu, Emily; Mutua, Sylvia; Kotes, Emma; Shults, Justine; et al. (Molecular Genetics & Metabolism, 2024)