Journal
MOLECULAR GENETICS AND METABOLISM
Volume 88, Issue 3, Pages 208-215Publisher
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.ymgme.2006.01.010
Keywords
Gaucher disease; Fabry disease; enzyme replacement; substrate reduction; cost; health care
Funding
- NIDDK NIH HHS [DK061370-03] Funding Source: Medline
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Potential treatment for lysosomal diseases now includes enzyme replacement therapy, substrate reduction therapy, and chaperone therapy. The first two of these have been implemented commercially, and the spectrum of diseases that are now treatable has expanded from Gaucher disease to include several other disorders. Treatment of these diseases is extremely costly. We explore some of the reasons for the high cost and discuss how, by proper selection of patients and appropriate dosing, the economic burden on society of treating these disease may be ameliorated, at least in part. However, the cost of treating rare diseases is a growing problem that society needs to address. (c) 2006 Elsevier Inc. All rights reserved.
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