Astellas details potential reason behind deaths in rare disease gene therapy trial
Four boys with a rare and often severe genetic muscle disease died in 2020 and 2021 after receiving a one-time gene therapy from Astellas Pharma, and a new study seeks to explain what caused their deaths.
In the study published Wednesday in Lancet Neurology, researchers detailed a potential explanation for the liver toxicity that led to the deaths of the four boys and depicted the striking benefits of the X-linked myotubular myopathy (XLMTM) gene therapy in the 20 children who survived.
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