Etuvetidigene Autotemcel for the Treatment of Wiskott-Aldrich Syndrome. (PubMed, N Engl J Med) - Sep 24, 2026 - P1/2, P3 | "Our results show that the effects of etu-cel are consistent with a sustained clinical benefit in persons with Wiskott-Aldrich syndrome. (Funded by Fondazione Telethon and others; TIGET-WAS and OTL-103-4 ClinicalTrials.gov numbers, NCT01515462 and NCT03837483, respectively.)."
Journal • Gene Therapies • Hematological Disorders • Immunology • Infectious Disease • Oncology • Primary Immunodeficiency
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; Alessandro Aiuti; Alessia Orsini; Carmen Caputo; Celeste Scotti; Daniele Canarutto; Davide Sala; Elena Tomasetto; Eugenio Montini; Fabio Ciceri; Federica Barzaghi; Federica Miotto; Federico Fraschetta; Francesca Ferrua; Francesca Fumagalli; Francesca Tucci; Giulia Consiglieri; Ilaria Monti; Koen van Rossem; Laura Castagnaro; Luigi Naldini; Maddalena Migliavacca; Maria Ester Bernardo; Maria Pia Cicalese; Mariam Datukishvili; Matias Soncini; Matteo Doglio; Paolo Silvani; Raffaella Milani; Sabina Cenciarelli; Salvatore Recupero; Sean Russell; Shanmuganathan Chandrakasan; Simona De Gregori; Stefania Galimberti; Stefania Giannelli; Stefano Zancan; Suhag Parikh; Valeria Calbi; Vera Gallo
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