FABRY DISEASE

This study looked at the long-term safety of a drug called migalastat in 16 people over age 12 with Fabry disease who have certain genetic changes. Fabry disease is a rare inherited condition that can cause pain, organ damage, and other serious problems. The goal was to see if ta…

This study tested a new enzyme replacement drug (AGA BETA BS) in 20 adults with Fabry disease who were already stable on the standard treatment, Fabrazyme. The goal was to see if switching to the new drug kept their disease marker (Lyso-Gb3) at similar levels over 6 months. The t…

This study looked at the long-term safety of a drug called pegunigalsidase alfa in adults with Fabry disease, a rare genetic disorder. 97 people who had already completed earlier studies received the drug every two weeks. The main goal was to check for side effects over time, and…

This study tested a new gene therapy called ST-920 in 36 adults with Fabry disease. The treatment uses a harmless virus to deliver a working gene that helps the body produce an enzyme it is missing. The goal was to see if a single intravenous dose is safe and tolerable, and wheth…

This study looked at how physical activity, sleep, and quality of life are connected in people with Fabry disease. About 100 adults in the UK wore a clinical-grade device and filled out questionnaires. The goal was to understand day-to-day challenges and mental health effects, si…

This study looked at 409 Portuguese adults with unexplained heart muscle disease (cardiomyopathy) to see how many had Fabry disease, a rare genetic condition. Researchers tested patients with different types of heart problems, such as thickened or weakened heart muscle. The goal …