FABRY DISEASE

This study looked 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. The 29 participants had already completed a previous study and received the drug every 4 weeks. Resear…

This study tested the safety and effectiveness of Fabrazyme, an enzyme replacement therapy, in 22 Chinese patients with Fabry disease. Fabry disease is a rare genetic disorder that causes pain, organ damage, and other serious symptoms. Patients received Fabrazyme infusions for 54…

This study tested whether a new, similar version of the drug agalsidase beta (called AGA BETA BS) works as well as the standard drug Fabrazyme in people with Fabry disease. Twenty adults who were already stable on Fabrazyme switched to the new drug for 6 months to a year. The mai…

This study looked at the long-term safety of a drug called pegunigalsidase alfa for adults with Fabry disease, a rare genetic disorder. A total of 97 people who had completed earlier studies received the drug through an IV every two weeks. The main goal was to track any side effe…

This study tested a new gene therapy called ST-920 for people with Fabry disease, a rare genetic condition. The therapy uses a harmless virus to deliver a working gene that helps the body produce an enzyme it's missing. 36 adults received a single intravenous dose and were follow…

This study looked at the long-term safety and effectiveness of the drug migalastat in 16 people over age 12 with Fabry disease who have certain genetic variants. Fabry disease is a rare genetic disorder that can cause pain, organ damage, and other serious problems. The goal was t…

This study aimed to find a better way to detect Fabry disease early by using computer programs that analyze hospital records from the last 10 years. Researchers looked at data from 50 patients to develop and test an algorithm that ranks people by how likely they are to have the d…

This study looked at how much time and money it takes for Fabry disease patients and their caregivers to receive enzyme replacement therapy. Researchers watched 82 adults during their regular infusions to measure the work for healthcare providers and the impact on patients' daily…

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 activity tracker and filled out questionnaires. The goal was to understand if low activity or poor sleep relates to …

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. This is an early-stage study that does not directly treat patients with…

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…