|
|
|
|
Fabry disease is a metabolic disorder without a specific treatment, caused by a deficiency of the lysosomal enzyme x-galactosidase A (x-gal A). Most patients experience debilitating neuropathic pain and premature mortality because of renal failure, cardiovascular disease, or cerebrovascular disease. Intravenous infusions of x-gal A are safe and have widespread therapeutic efficacy in Fabry disease. |
|