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Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy

Adeno-associated virus (AAV) gene therapies are highly promising, such as the onasemnogene abeparvovec (Zolgensma) in spinal muscle atrophy (SMA). We report the first case of fatal systemic thrombotic microangiopathy (TMA) following onasemnogene abeparvovec in a 6-month-old child with SMA type 1, ca...

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Detalles Bibliográficos
Autores principales:	Guillou, Julia, de Pellegars, Alice, Porcheret, Florence, Frémeaux-Bacchi, Véronique, Allain-Launay, Emma, Debord, Camille, Denis, Manon, Péréon, Yann, Barnérias, Christine, Desguerre, Isabelle, Roussey, Gwenaëlle, Mercier, Sandra
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Hematology 2022
Materias:	Stimulus Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9327533/ https://www.ncbi.nlm.nih.gov/pubmed/35584395 http://dx.doi.org/10.1182/bloodadvances.2021006419

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