This study investigates pregnancy outcomes in women with Pompe disease. This study is part of a larger registry that tracks the health of women with Pompe disease during pregnancy, whether or not they are receiving specific treatments like enzyme replacement therapy (ERT).Participants will undergo clinical assessments and receive standard care …
This study investigates Pompe disease, focusing on muscle function impacts due to glycogen buildup. The purpose of this observational study is to track the natural history and outcomes of Pompe disease in patients worldwide, regardless of treatment status. An observational study involves collecting data by observing participants receiving routine care, …
This study investigates the safety of an investigational medication when used by women during pregnancy and lactation. The purpose is to collect data on the effects of the medication on pregnancy outcomes, maternal health, and the developing child. Outcomes in infants exposed to the medication will be assessed, including their …
