Enzyme replacement is mostly done for:
Ans. (c) Gaucher's diseaseRef: Harper's Biochemistry, 30th ed. pg. 251Management of Gaucher's Disease* Regular, intravenous enzyme therapy is currently the treatment of choice in affected patients and is highly efficacious and safe in diminishing the hepatosis and improving bone marrow involvement and hematologic findings.* Symptomatic management of the blood cytopenias and joint replacement continue to have important roles in management* The bone disease is decreased but not eliminated by enzyme therapy. Adults benefit from adjunctive treatment with bisphosphonates to improve bone mass.
Niemann pick's
Gangliosidosis
Gaucher's disease
Phenylketonuria
Lipids