This Plain Language Summary of publication article (PLSP) from Future Rare Diseases explains how Fabry disease is caused by a missing or malfunctioning enzyme called alpha-Gal A. Agalsidase beta, an enzyme replacement therapy, is used to treat the disease. Researchers analyzed data from the Fabry Registry on people who began treatment between ages 5 and 30. They examined kidney function, heart health, and symptoms like stomach aches, diarrhea, pain in the hands and feet, and pain crises.

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This PLSP is based on an article called ‘Clinical outcomes among young patients with Fabry disease who initiated agalsidase beta treatment before 30 years of age: An analysis from the Fabry Registry’ and was published in Molecular Genetics and Metabolism.

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