Wikipedia

A gene therapy study, halted when four children with a rare neuromuscular disorder died after treatment, showed enough promise to merit finding a path forward, according to the medicine’s manufacturer.

The treatment, made by the Japanese pharmaceutical company Astellas, led to severe and fatal liver problems for four of the 24 treated children with X-linked myotubular myopathy, or XLMTM, a genetic disease that severely degrades muscle function and kills most patients before the age of 10. Three died in 2020; the fourth died a year later.

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In an analysis of its clinical trial, published in Lancet Neurology on Wednesday, Astellas observed dramatic benefits in the study’s surviving patients and a potential explanation for the deaths that derailed the trial. The hope, for Astellas and for families affected by XLMTM, is that the company can find a safe way to proceed and convince the Food and Drug Administration to allow it to resume development.

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