A novel patient-Centered approach to clinical trial readiness in rare diseases: Application in Aicardi-Goutières Syndrome (AGS)
Anjana Sevagamoorthy(Children's Hospital of Philadelphia), Adeline Vanderver(Children's Hospital of Philadelphia), Cherié Butts(Biogen (United States)), Jessica Galli(Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia), Samuel A. Pierce(Children's Hospital of Philadelphia), Marie Hully(Hôpital Necker-Enfants Malades), Allan M. Glanzman(Boston Children's Hospital), Ylenia Vaia, Davide Tonduti(Luigi Sacco Hospital), Devon Cordova(ME Association), Elise L. Townsend(MGH Institute of Health Professions), Francesco Gavazzi(Children's Hospital of Philadelphia), Melissa Raspa(RTI International), Evangeline Wassmer(Birmingham Children's Hospital), Elisa Maria Fazzi(Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia), Pamela Ventola(Yale University), Ann T. Harrington(Children's Hospital of Philadelphia), Sara B. DeMauro(Children's Hospital of Philadelphia), Elizabeth M. Berry Kravis(Rush University), Peter Hong(Children's Hospital of Philadelphia), Michelle Fitzgerald(Children's Hospital of Philadelphia), Zarrin Tashnim(Children's Hospital of Philadelphia), Min Ae Lee-Kirsch(University Hospital Carl Gustav Carus), Despina Eleftheriou(University College London), Anne Wheeler(RTI International), Tina Duong(Stanford Medicine), Stacy Cusack(Children's Hospital of Philadelphia), Shanice Beerepoot(Amsterdam Neuroscience), Laura A. Adang(Children's Hospital of Philadelphia), Simona Orcesi(Fondazione Istituto Neurologico Nazionale Casimiro Mondino)
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