Access to gene therapy for rare diseases when commercialization is not fit for purpose. Correspondence from Claire Booth GreatOrmondSt and the AGORA initiative.
Thomas Fox, Adrian Thrasher & Claire Booth
Biomedical Innovation Unit, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas , Centro Investigación Biomédica en Red de Enfermedades Raras and IIS Fundación Jiménez Díaz, Madrid, SpainDivision of Immunology and Allergy, University Hospital of Lausanne and Lausanne University, Lausanne, SwitzerlandSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute and Vita-Salute San Raffaele University, Milan,...
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