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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