FABRY DISEASE

This study tested a new gene therapy, 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 infusion is safe and can reduce harmful buildup in the…

This study looked at the long-term safety of a drug called migalastat in 16 people over age 12 with Fabry disease, a rare genetic disorder. Participants had already completed an earlier study and continued taking migalastat to see if it was safe and how it affected kidney functio…

This study tested a new enzyme replacement drug (AGA BETA BS) for Fabry disease, a rare genetic disorder. It involved 20 adults 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 s…

This study looks at the long-term safety and effectiveness of a drug called pegunigalsidase alfa for adults with Fabry disease, a rare genetic condition that can damage organs. Participants, who had already completed a previous study, receive an infusion of the drug every 4 weeks…

This study looked at the long-term safety of a drug called pegunigalsidase alfa in 97 adults with Fabry disease. Participants received the drug through a vein every two weeks. The main goal was to track any side effects over time. This was an extension study for people who had al…

This study tested the safety and effectiveness of Fabrazyme, an enzyme replacement therapy, in 22 Chinese patients with Fabry disease. Participants received the drug for 54 weeks to see if it could control disease symptoms and reduce harmful substances in the blood. The goal was …

This study aimed to find a better way to detect Fabry disease early by using computer programs that analyze hospital records. Researchers looked at data from the past 10 years to create an algorithm that ranks patients by how likely they are to have the disease. No new treatments…

This study looked at hospital bills for over 15,000 people with cardiomyopathy, a heart muscle disease. Researchers collected information on what patients spent during hospital stays to understand the real-world costs. The goal is to help doctors and hospitals better manage expen…

This study looked at how enzyme replacement therapy (ERT) for Fabry disease affects the daily lives of patients, their caregivers, and healthcare providers. Researchers measured the time spent preparing and giving the treatment, as well as its impact on patient quality of life, f…

This study tested whether a special MRI scan (T1 mapping) can measure kidney scarring in people with Fabry disease. Researchers compared 70 adults with Fabry disease to healthy controls to see if the scan could detect early kidney damage. The goal is to improve how doctors decide…

This study tested a new version of the drug agalsidase beta (made by Biosidus) against the approved version (Fabrazyme) in 24 healthy men. The goal was to see if the new version behaves similarly in the body. Participants received a single dose by IV, and researchers measured dru…

This study monitored 100 adults with Fabry disease in the UK to understand how their daily physical activity, sleep, and quality of life are connected. Participants wore a clinical-grade device and filled out questionnaires about their mental health and well-being. The goal was t…

This study looked at 409 adults in Portugal who had heart muscle diseases (cardiomyopathies) with no clear cause. The goal was to find out how many of them actually had Fabry disease, a rare genetic condition that can affect the heart. Researchers tested patients with different t…