FABRY DISEASE

This trial tested a new gene therapy called ST-920 for 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 with Fabry disease received a single intravenous dose and were…

This completed Phase 4 study tested Fabrazyme (agalsidase beta) in 22 Chinese participants with Fabry disease, a rare genetic disorder. The treatment replaces a missing enzyme to help control the disease. Researchers monitored safety and changes in disease markers over 54 weeks.

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

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. Participants received an infusion every four weeks for up to several years. The goal was to see if the drug is safe over time…

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 tested whether a special MRI scan called T1 mapping can detect kidney damage in people with Fabry disease, a rare genetic disorder. Researchers compared MRI results from 70 adults with and without Fabry disease. The goal was to see if this non-invasive scan could help …

This study collected information from over 15,000 people hospitalized with cardiomyopathy, a heart muscle disease. Researchers looked at how much these hospital stays cost and what the money was spent on. The goal was to understand the real-world financial impact and help manage …

This early study tested whether a new version of the enzyme replacement drug agalsidase beta (made by Biosidus) works similarly to the approved drug Fabrazyme. Twenty-four healthy men received a single infusion of one of the two drugs. Researchers measured how the drug moved thro…

This study aims to develop a computer algorithm that can detect Fabry disease earlier by analyzing hospital electronic health records. Researchers will look at data from the past 10 years to see if they can identify patients with Fabry disease using a ranking system. No drugs or …

This study looked for Fabry disease, a rare genetic disorder, in 409 Portuguese adults with unexplained heart muscle diseases. Researchers tested patients with different types of cardiomyopathy to see how many actually had Fabry disease. The goal was to better understand how comm…

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 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…