juMPStart: Phase 1, Open-Label, Dose-Escalation Safety and Efficacy Gene Therapy Study Evaluating HMI-203 in Adults with MPS II

juMPStart: Phase 1, Open-Label, Dose-Escalation Safety and Efficacy Gene Therapy Study Evaluating HMI-203 in Adults with MPS II

Shioshita G, Henrick J, Verhofstadt C, et al. ACMG 2023.