Table 2.

Enzyme replacement therapy.

Disease	Enzyme	Therapeutic responses
1. Gaucher	• β-glucosidase	• Improved blood counts, reduction of liver and spleen volume, improved bone density at the lumbar spine
2. Fabry	• α-galactosidase A	• Partially mitigates neuropathic pain and gastrointestinal discomfort, modifies course of renal disease in mild to moderately CKD, reduces left ventricular hypertrophy; does not appear to significantly alter incidence of stroke
3. MPS disorders		• Reduced urinary GAG excretion; reduced liver and spleen volume, improvement in FVC, increased distance travelled in time walk
— Type I	• α-L-iduronidase
— Type II	• Iduronate-2-sulfatase
— Type VI	• Arylsufatase B
4. Pompe	• α-glucosidase	• Resolution of cardiomyopathy (infantile-onset cases); stabilization or improvement in muscle strength; respiratory improvement

CKD, chronic kidney disease; GAG, glycosaminoglycans; FVC, forced vital capacity.