• Ph 3 STR1VE data show prolonged event-free survival, early and rapid increases in CHOP-INTEND and significant milestone achievement in SMA Type 1, consistent with START trial • First-in-human biodistribution data show transduction in intended CNS targets and widespread SMN expression comparable to tissue from unaffected control • More than 150 patients treated with Zolgensma, only 5% of screened patients up to 5 years old excluded due to AAV9 antibody titers greater than 1:50
April 17, 2019
· 12 min read