Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series.
case_series · Level IV
Where this comes from
- Record sourced from PubMed, PMID 33259859.
- Also identified by DOI 10.1016/j.jpeds.2020.11.054.
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Abstract
Spinal muscular atrophy is treated with onasemnogene abeparvovec, which replaces the missing survival motor neuron 1 gene via an adeno-associated virus vector. As of July 1, 2020, we had identified 3 infants who developed thrombotic microangiopathy following onasemnogene abeparvovec. Early recognition and treatment of drug-induced thrombotic microangiopathy may lessen mortality and morbidity.
Medical subject headings
- Biological Products
- Muscular Atrophy, Spinal
- Recombinant Fusion Proteins
- Thrombotic Microangiopathies