Enzyme Replacement Therapy Articles & Analysis
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(Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today reported financial results for the first quarter ended March 31, 2022 and provided a business update. ...
” As of the cutoff date, elevated α-Gal A activity was maintained for the four patients treated in the first two dose cohorts (0.5e13 vg/kg and 1e13 vg/kg) ranging from 3-fold to 15-fold above mean normal at last measurement. For the two patients on enzyme replacement therapy (ERT), α-Gal A activity measured at ERT trough was ...
Activity of 2-fold to 15-fold above mean normal was observed at last measurement as of the cutoff date. Withdrawal from enzyme replacement therapy (ERT) has taken place for one patient and is planned for the other patient on ERT, based on the stability of their α-Gal A activity following treatment. ...