
Sanofi-Genzyme
Contact: Amy Fitzgerald
330-814-4451

Fabry disease is an inherited Lysosomal Storage Disorder (LSD) caused by deficient activity of the enzyme α-galactosidase A. Impaired degradation and subsequent accumulation of α-galactosidase A substrates in lysosomes causes multi-systemic, progressive and life-threatening disease.1,2 Secondary disease processes eventually lead to renal failure, cardiac failure or cerebrovascular complications.