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Article

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

18 May 2022

Abstract excerpt

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, carrying a potential genetic predisposition in the complement factor I gene. Other cases of TMA have recently been...

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PMID: 35584395
DOI: 10.1182/bloodadvances.2021006419

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