AI Article Synopsis
- Spinal muscular atrophy treatment includes onasemnogene abeparvovec, which delivers a replacement gene using a virus vector.
- Since July 1, 2020, three infants were found to have developed thrombotic microangiopathy after receiving this treatment.
- Early detection and intervention for this condition might reduce the associated risks of death and serious complications.
Article 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.
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| http://dx.doi.org/10.1016/j.jpeds.2020.11.054 | DOI Listing |
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