Current Research Studies

A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations

HLB001_Ph1

What is the goal of the study?

To learn more about this study, please email the study team member listed below.

Who can participate in the study?

Please contact the study team listed below to learn more.

Study Team: