Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

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 three infants who developed thrombotic microangiopathy (TMA) following onasemnogene abeparvovec . Early recognition and treatment of drug-induced TMA may lessen mortality and morbidity.

Source: The Journal of Pediatrics - November 27, 2020 Category: Pediatrics Authors: Deepa H. Chand, Craig Zaidman, Kapil Arya, Rachel Millner, Michelle A. Farrar, Fiona E. Mackie, Natalie L. Goedeker, Vikas R. Dharnidharka, Raja Dandamudi, Sandra P. Reyna Tags: Brief Reports Source Type: research