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 Fabry disease, focusing on observing and collecting data on individuals diagnosed with the condition, regardless of their treatment status, to better understand the disease's progression and impact.The study aims to gather information on the safety and effectiveness of treatments for Fabry disease, as well as to track …
