Real-world Multidisciplinary Outcomes of Onasemnogene Abeparvovec Monotherapy in Patients with Spinal Muscular Atrophy Type 1: Experience of the French Cohort in the First Three Years of Treatment Isabelle Desguerre , Rémi Barrois , Frédérique Audic , Christine Barnerias , Brigitte Chabrol , Jean Baptiste Davion , Julien Durigneux , Caroline Espil-Taris , Marta Gomez-Garcia de la Banda , Marine Guichard , Arnaud Isapof , Marie Christine Nougues , Vincent Laugel , Laure Le Goff , Sandra Mercier , Anne Pervillé , Christian Richelme , Marie Thibaud , Catherine Sarret , Cyril Schweitzer , Hervé Testard , Valérie Trommsdorff , Catherine Vanhulle , Ulrike Walther-Louvier , Cécilia Altuzarra , Mondher Chouchane , Juliette Ropars , Susana Quijano-Roy , Claude Cances Orphanet Journal of Rare Diseases（2024）
关键词
Spinal muscular atrophy, Gene transfer therapy, Real-world outcome
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