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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