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. 1975 Apr;34(5):1310-5.

Investigations in enzyme replacement therapy in lipid storage diseases

R O Brady, P G Pentchev, A G Gal
  • PMID: 804420

Investigations in enzyme replacement therapy in lipid storage diseases

R O Brady et al. Fed Proc. 1975 Apr.

Abstract

Enzyme replacement appears to offer much promise as specific therapeutic procedures for patients with Fabry's disease and Gaucher's disease. However, enzyme replacement in patients with Tay-Sachs disease and other heritable metabolic disorders where the central nervous system is affected will require first the development of effective methods for the delivery of exogenous enzymes to the brain; such methods are not yet available.

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