Current Research Studies

A prospective observational study to describe clinical outcomes of alglucosidase alfa treatment in patients ≤6 months of age with infantile-onset Pompe disease (IOPD)

OBS17003 - babyCOMET Comparator

Condition(s): Other
Phase: N/A
Clinicaltrials.gov ID: NCT04848779

What is the goal of the study?

An observational study that follows IOPD patients who start Lumizyme, a standard of care treatment for Pompe disease, at under 6 months of age.

Who can participate in the study?

Please contact the study team listed below to learn more.

Study Team:

PI (Study Doctor): Sihoun Hahn
Study Team Member: Phi Duong, Phi.Duong@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.