This study investigates pregnancy outcomes in women with Pompe disease. Pompe disease is a rare genetic disorder that affects the body's ability to break down a complex sugar called glycogen, leading to muscle weakness and other health issues. The study does not involve any experimental treatments. Instead, it focuses on …
This study investigates Pompe disease, a rare genetic disorder that affects the body's ability to break down a complex sugar called glycogen. The purpose of this study is to understand how Pompe disease progresses over time and how it varies among patients. This includes looking at patients who receive treatment …
This study investigates the effects of an investigational medication on infants with a condition called Infantile-Onset Pompe Disease (IOPD). IOPD is a rare genetic disorder where the body cannot break down certain complex sugars due to a missing or non-working enzyme. This study aims to understand how routine use of …
This study investigates the safety of an investigational medication taken by women during pregnancy and breastfeeding. The study aims to understand any risks this medication may have on pregnancy, as well as any complications or side effects it may cause in the developing baby, newborn, and infant. The study will …
