Treatment
There are no cures for lysosomal storage diseases and treatment is mostly symptomatic, although bone marrow transplantation and enzyme replacement therapy (ERT) have been tried with some success. In addition, umbilical cord blood transplantation is being performed at specialized centers for a number of these diseases. In addition, substrate reduction therapy, a method used to decrease the accumulation of storage material, is currently being evaluated for some of these diseases. Furthermore, chaperone therapy, a technique used to stabilize the defective enzymes produced by patients, is being examined for certain of these disorders. The experimental technique of gene therapy may offer cures in the future.
Read more about this topic: Lysosomal Storage Diseases
Famous quotes containing the word treatment:
“The treatment of the incident of the assault upon the sailors of the Baltimore is so conciliatory and friendly that I am of the opinion that there is a good prospect that the differences growing out of that serious affair can now be adjusted upon terms satisfactory to this Government by the usual methods and without special powers from Congress.”
—Benjamin Harrison (1833–1901)
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—C. John Sommerville (20th century)
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—Hippocrates (c. 460–c. 370 B.C.)