Treatment options for lysosomal storage disorders: developing insights.
van Gelder CM, Vollebregt AA, Plug I, van der Ploeg AT, Reuser AJ
Expert opinion on pharmacotherapy, 2012 Nov
Abstract
Lysosomal storage disorders (LSDs) are clinically heterogeneous disorders that result primarily from lysosomal accumulation of macromolecules in various tissues. LSDs are always progressive, and often lead to severe symptoms and premature death. The identification of the underlying genetic and enzymatic defects has prompted the development of various treatment options.