UPDATED: Fourth boy dies in Astellas-Audentes gene therapy trial, raising fresh fears for the field
A fourth boy has died in the Astellas-Audentes gene therapy trial for a rare muscle-wasting disease, the company announced Tuesday morning.
The death comes a little over a year after the first of three boys with myotubular myopathy, or XLMTM, died after receiving the therapy and suffering liver failure. The company was cleared to resume studies in December after laying out a plan to give patients a much lower dose and enrolled their first new volunteer over the summer.
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