Plain Language Summary: Looking at treatment outcomes in people with Fabry disease who started agalsidase beta before the age of 30 years

What did this study look at? An enzyme called alpha-galactosidase (alpha-Gal A) does not work properly or is absent in people with Fabry disease. Agalsidase beta (an enzyme replacement therapy) is a treatment for Fabry disease that substitutes for the non-working or absent alpha-Gal A enzyme. Using information from the Fabry Registry on young people who started agalsidase beta treatment between 5 and 30 years of age, researchers looked at the following over time: Kidney function decline.Heart muscle health.Fabry-related symptoms: ○ Stomach aches. ○ Diarrhoea. ○ Tingling or burning pain in hands and feet. ○ Pain crises (attacks of severe pain, also called Fabry crises).Characteristics of the people studied were: Males were younger than females when: ○ Symptoms first started. ○ Agalsidase beta treatment was first started.