Morbus Fabry is a rare lysosomal storage disease in which an enzyme defect prevents the breakdown of certain metabolic products. These accumulate in cells and damage various organs such as the heart, kidneys and nervous system.

Treatment
In the past, treatment was limited to alleviating symptoms. Today, two causal treatment approaches are available:

1. Enzyme replacement therapy (ERT)
Since 2001, the missing enzyme alpha-galactosidase A can be replaced by biotechnologically produced preparations.

• Infusion every two weeks at the doctor's office or at home by a specialist

• Regular medical check-ups required

• Lifelong treatment to prevent new deposits

2. Chaperone therapy
Available for certain gene mutations since 2017.

• Stabilizes the body's own faulty enzyme so that it can function again

• Take one capsule every two days (2 hours apart from meals)

• Slows the progression of the disease and relieves symptoms

Note: Further information and suitable treatment options can be obtained from your doctor.