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

Condition(s): Other
Clinicaltrials.gov ID: NCT04581785

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:

PI (Study Doctor): Angela Sun
Study Team Member: Hayden Vreugdenhil, Hayden.Vreugdenhil@seattlechildrens.org

Seattle Children’s complies with applicable federal and other civil rights laws and does not discriminate, exclude people or treat them differently based on race, color, religion (creed), sex, gender identity or expression, sexual orientation, national origin (ancestry), age, disability, or any other status protected by applicable federal, state or local law. Financial assistance for medically necessary services is based on family income and hospital resources and is provided to children under age 21 whose primary residence is in Washington, Alaska, Montana or Idaho.