FABRY DISEASE

This study tested a new, similar version of the drug agalsidase beta (called AGA BETA BS) in 20 people with Fabry disease who were already doing well on the standard drug Fabrazyme. The goal was to see if switching to the new drug kept their disease marker (Lyso-Gb3) at the same …

This study tested a drug called Fabrazyme in 22 Chinese patients with Fabry disease, a rare genetic condition that causes pain and organ damage. Patients received enzyme replacement therapy for 54 weeks to see if it was safe and helped control the disease. The main focus was on s…

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 better ways to detect Fabry disease early by analyzing hospital records from the past 10 years. Researchers used natural language processing, a type of computer analysis, to create a ranking algorithm that identifies patients most likely to have the disea…

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…

This study looked at how physical activity, sleep, and quality of life are connected in people with Fabry disease. Researchers used a wearable device and questionnaires to track 100 adults in the UK. The goal was to understand day-to-day challenges that standard medical tests mig…

This study looked at how much time healthcare professionals spend preparing and giving enzyme replacement therapy to adults with Fabry disease, and how the treatment affects patients and their caregivers. Researchers measured tasks like setting up the infusion and checking on pat…

This study tested two versions of the drug agalsidase beta (one from Biosidus, one from Sanofi) in 24 healthy men. Participants received a single dose by IV, and researchers measured how much drug got into the blood and how active it was. The goal was to see if the two versions w…