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 how it progresses over time in individuals diagnosed with the condition.The study aims to understand the variability and progression of Pompe disease. It seeks to gather information to aid in developing patient care recommendations and evaluating the effectiveness of long-term treatments.Who can participate: …
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 …