Gaucher's (pronounced go-SHAYZ) disease is a rare but a most common lysosomal storage disorder and genetic disorder affecting Jewish people of Eastern European ancestry.
Gaucher's disease is fatal condition wherein a defective form of GC is produced. GC is a critical enzyme that breaks down a fatty substance called glucosylceramide.
Current available treatment of Gaucher’s disease is an intravenous treatment (enzyme replacement therapy) that must be administered for life but costs up to $750,000 annually per patient
New research findings reported in the May issue of ACS Chemical Biology sheds a light into the expensive treatment of Gaucher’s disease.
The new study involves an oral treatment consisting of “chemical chaperones” (small molecules: natural and synthetic substances with a low molecular weight) that can partially correct the genetic defect responsible for most cases of Gaucher's disease. This oral treatment could cut the expensive treatment costs of the disease by 100-fold.
Read more at EurekAlert.
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