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BioWorld - Saturday, July 11, 2026
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Home » Fourth child dies on Astellas’ gene therapy trial, after FDA advisers cite liver safety issues
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Fourth child dies on Astellas’ gene therapy trial, after FDA advisers cite liver safety issues

Sep. 14, 2021
By Richard Staines
A fourth child has died after developing liver complications on a trial of Astellas Pharma Inc.’s gene therapy for rare neuromuscular disease, after FDA advisers noted the problems on the ASPIRO study in a discussion on gene therapy safety. Last week, Astellas announced that it had stopped dosing on ASPIRO after a safety issue involving liver function emerged in the trial of the gene therapy AT-132, aimed at the life-threatening rare disease X-linked myotubular myopathy.
BioWorld Clinical Regulatory Neurology/psychiatric Gene therapy FDA

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