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Lancet (London, England)|January 23, 2022
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded accessFrancesca Fumagalli, Valeria Calbi, Maria Grazia Natali Sora, et al.The New England Journal of Medicine|November 17, 2021
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler SyndromeBernhard Gentner, Francesca Tucci, Stefania Galimberti, et al.Nature Medicine|February 15, 2024
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiencyMaddalena Migliavacca, Federica Barzaghi, Claudia Fossati, et al.The New England Journal of Medicine|April 23, 2025
Long-Term Effects of Atidarsagene Autotemcel for Metachromatic LeukodystrophyFrancesca Fumagalli, Valeria Calbi, Vera Gallo, et al.Frontiers in Immunology|March 31, 2018
Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-Kinase δ Syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome RegistryMaria Elena Maccari, Hassan Abolhassani, Asghar Aghamohammadi, et al.Pageof 4