This study investigates the safety and efficacy of a one-time intravitreal injection of an investigational medication for participants with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). The study is divided into two parts to assess different aspects of the treatment.Participants will receive a one-time injection into the eye, …
This study investigates pregnancy outcomes in women with Pompe disease. The study is observational, meaning it involves monitoring and collecting data on pregnant women with Pompe disease, whether or not they are receiving enzyme replacement therapy (ERT) or other treatments. The aim is to gather information on pregnancy complications and …
This study investigates Pompe disease, a rare genetic disorder. The purpose of this observational study is to track the disease's natural history and outcomes in patients, both those receiving treatment and those who are not. An observational study is a type of research study where data is collected by observing …
This study investigates the safety of an investigational medication when used by women during pregnancy and/or lactation. The purpose is to assess any risks associated with the investigational medication on pregnancy, as well as potential complications and adverse effects in the developing fetus, neonate, and infant.Participants will have their data …
