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
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.