Amy Y. Chen

This study evaluates the 12-month outcomes of vesemnogene lantuparvovec, an intrathecal gene therapy for SMA in children under 24 months in LMICs, showing safety and motor milestone improvements.

This paper reports on a phase 1 trial of vesemnogene lantuparvovec, an affordable gene therapy for SMA, showing safety and improved motor milestones in young children.

The paper surveys lab-made plasmids globally and finds nearly half contain errors, with 40% of transfer plasmids having mutations in ITR regions due to instability.