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 Modesti, Emily Yu, Sylvia Mutua, Emma Kotes, Justine Shults, Ariel Vincent, Lisa Emrick, Stephanie Keller, Keith Van Haren, Sarah Woidill, Isabella Peixoto de Barcelos, Amy Pizzino, Johanna Schmidt, Florian Eichler, Ali Fatemi, Adeline Vanderver