Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach
Laura Ann Adang(Children's Hospital of Philadelphia), Adeline Vanderver(Children's Hospital of Philadelphia), Anjana Sevagamoorthy(Children's Hospital of Philadelphia), Florian Eichler(Harvard University), Sarah Woidill(Children's Hospital of Philadelphia), Stephanie Keller(Children's Healthcare of Atlanta), Isabella Peixoto de Barcelos(Children's Hospital of Philadelphia), Joshua L. Bonkowsky(Intermountain Healthcare), Francesco Gavazzi(Children's Hospital of Philadelphia), Keith Van Haren(Stanford University), Omar Sherbini(Children's Hospital of Philadelphia), Jamie L. Fraser(Children's National), Nicholson Modesti(Children's Hospital of Philadelphia), Ariel Vincent(Children's Hospital of Philadelphia), Lisa Emrick(Baylor College of Medicine), Amy Pizzino(Children's Hospital of Philadelphia), Russel D'Aiello(Children's Hospital of Philadelphia), Sylvia Mutua(Children's Hospital of Philadelphia), Emma Kotes(Children's Hospital of Philadelphia), Johanna Schmidt(Counseling Center), Justine Shults(Children's Hospital of Philadelphia), Emily Yu(Children's Hospital of Philadelphia), Ali Fatemi(Kennedy Krieger Institute)
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