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Treatments for lysosomal storage disorders
Biochemical Society Transactions, 2010There are over 70 human diseases that are caused by defects in various aspects of lysosomal function. Until 20 years ago, the only specific therapy available for lysosomal storage disorders was allogeneic haemopoietic stem cell transplantation. Over the last two decades, there has been remarkable progress and there are now licensed treatments for seven
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Lysosomal Storage Disorders and Iron
2013Lysosomal storage disorders are a group of about 50 rare metabolic diseases that result from defects in lysosomal function. The majority is recessively inherited and caused by mutations in genes encoding lysosomal proteins as the basis for its pathobiology.
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PSEUDODEFICIENCIES IN LYSOSOMAL STORAGE DISORDERS
The Lancet, 1985J, Zlotogora, G, Bach
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