Cross-Correction in HSC Gene Therapy for Metachromatic Leukodystrophy
Vasco Meneghini, Valeria Calbi, Filippo Casalini, Elisabeth Mangiameli et autres
0 citationsNew England Journal of Medicine
Francesca Fumagalli
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71Publications signalées
2989Citations signalées
5Affiliations récentes
Institutions déclarées
Domaines associés
Lysosomal Storage Disorders ResearchRNA regulation and diseaseVirus-based gene therapy researchGlycogen Storage Diseases and MyoclonusTrypanosoma species research and implications
Publications récentes
European expert recommendations for comprehensive pre-treatment, treatment-phase and post-treatment care of patients with metachromatic leukodystrophy treated with autologous haematopoietic stem and progenitor cell gene therapy
Lucia Laugwitz, Francesca Fumagalli, Katharina Wehner, Pascal Martin et autres
1 citationEuropean Journal of Paediatric Neurology
Preliminary Data of the First Year of Newborn Screening for Metachromatic Leukodystrophy (MLD) in Lombardy
Alessandra Vasco, Andrea Meta, Clarissa Berardo, Davide Camerlengo et autres
0 citationsInternational Journal of Neonatal Screening
Lo screening neonatale per la leucodistrofia metacromatica: l'esempio toscano
Sabrina Malvagia, Alessandra Bettiol, Margherita Porcaro, Massimo MURA et autres
0 citationsBiochimica Clinica
Beyond ventricular fibrillation waveform analysis: a new era of personalised defibrillation
Francesca Fumagalli, Giuseppe Ristagno
1 citationResuscitation
Lentiviral hematopoietic stem and progenitor cell gene therapy for Wiskott-Aldrich Syndrome: Early access experience in Italian patients based on 648/1996 law
Sabina Cenciarelli, Stefania Giannelli, Federico Fraschetta, Carmela Caputo et autres
0 citationsBlood
Safety of Sodium-glucose Cotransporter 2 (SGLT2) Inhibitor “Dapagliflozin” after Cardiac Arrest and Cardiopulmonary Resuscitation: a Pilot Study
Daria De Giorgio, Marianna Cerrato, Francesca Callegari, Carlo Perego et autres
0 citationsResuscitation
Non-neurological, non-skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy (OTL-203) for Hurler syndrome
Francesca Tucci, María Luz Uria Oficialdegui, Giulia Consiglieri, Matilde Cossutta et autres
4 citationsMolecular Therapy
ARSA Variants Associated With Cognitive Decline and Long‐Term Preservation of Motor Function in Metachromatic Leukodystrophy
Shanice Beerepoot, Daphne H. Schoenmakers, Francesca Fumagalli, Samuel Groeschel et autres
2 citationsJournal of Inherited Metabolic Disease
Metachromatic Leukodystrophy
Marije A B C Asbreuk, Daphne H. Schoenmakers, Laura Adang, Shanice Beerepoot et autres
12 citationsNeurology
Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy
Daphne H. Schoenmakers, Marije A B C Asbreuk, Tamara J. Martin, Mareen R. Datema et autres
7 citationsEuropean Journal of Paediatric Neurology
Newborn Screening for Metachromatic Leukodystrophy in Tuscany: The Paradigm of a Successful Preventive Medicine Program
Sabrina Malvagia, Alessandra Bettiol, Margherita Porcaro, Massimo Mura et autres
3 citationsInternational Journal of Neonatal Screening
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