کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
1999130 1065842 2006 8 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Lysosomal storage diseases: Natural history and ethical and economic aspects
موضوعات مرتبط
علوم زیستی و بیوفناوری بیوشیمی، ژنتیک و زیست شناسی مولکولی زیست شیمی
پیش نمایش صفحه اول مقاله
Lysosomal storage diseases: Natural history and ethical and economic aspects
چکیده انگلیسی

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.

ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Molecular Genetics and Metabolism - Volume 88, Issue 3, July 2006, Pages 208–215
نویسندگان
,