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Reduction of large soft-tissue Gaucheromas with substrate reduction therapy

Neha Mahajan1, Mikako Warren2, Shoji Yano3

  • 1Department of Pediatrics, LAC+USC Medical Center, University of Southern California, Los Angeles, California.

Journal of Inherited Metabolic Disease
|November 10, 2019
PubMed
Summary

Soft-tissue masses in Gaucher disease are rare. Combining enzyme replacement therapy (ERT) with substrate reduction therapy effectively reduced large masses in a Gaucher disease type 3 patient.

Keywords:
Gaucher diseaseGaucheromaenzyme replacement therapy (ERT)substrate reduction therapy (SRT)

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