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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.

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Title: 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.
Authors: Desguerre, Isabelle1,2 (AUTHOR), Barrois, Rémi2 (AUTHOR) remi.barrois@aphp.fr, Audic, Frédérique3 (AUTHOR), Barnerias, Christine2 (AUTHOR), Chabrol, Brigitte3 (AUTHOR), Davion, Jean Baptiste4 (AUTHOR), Durigneux, Julien5 (AUTHOR), Espil-Taris, Caroline6 (AUTHOR), Gomez-Garcia de la Banda, Marta7 (AUTHOR), Guichard, Marine8 (AUTHOR), Isapof, Arnaud9 (AUTHOR), Nougues, Marie Christine9 (AUTHOR), Laugel, Vincent10 (AUTHOR), Le Goff, Laure11 (AUTHOR), Mercier, Sandra12 (AUTHOR), Pervillé, Anne13 (AUTHOR), Richelme, Christian14 (AUTHOR), Thibaud, Marie15 (AUTHOR), Sarret, Catherine16 (AUTHOR), Schweitzer, Cyril17 (AUTHOR)
Source: Orphanet Journal of Rare Diseases. 9/13/2024, Vol. 19 Issue 1, p1-11. 11p.
Database: Academic Search Ultimate
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ISSN:17501172
DOI:10.1186/s13023-024-03326-3