Words similar to gaucher

Example sentences for: gaucher

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This profitability has served as a stimulus for the development of enzyme replacement treatments for diseases less common and generally less responsive to treatment than Gaucher disease.
The development of enzyme replacement therapy for Gaucher disease, that is, exogenous administration of the missing enzyme, is a triumph of translational medicine.
For example, deficiencies of various enzymes responsible for lipid metabolism result in lipid accumulation in lysosomal storage diseases such as Niemann-Pick and Gaucher diseases [ 2 ] . Alternatively, modulation of lysosomal lumen pH may alter membrane trafficking and associated metabolic processes.
Type 1 Gaucher disease, the most common type, seems a particularly suitable target for enzyme replacement therapy because of the lack of central nervous system involvement (visceral damage in Gaucher disease is reversible whereas the brain damage usually is not).
Accordingly, many—possibly most—patients with Gaucher disease require no treatment.