Substrate deprivation therapy: a new hope for patients suffering from neuronopathic forms of inherited lysosomal storage diseases
Author:
Publisher
Springer Science and Business Media LLC
Subject
Genetics,General Medicine
Link
http://link.springer.com/content/pdf/10.1007/BF03195237.pdf
Reference32 articles.
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3. Beck M, 2007. New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy. Hum Genet 121: 1–22.
4. Bembi B, Marchetti F, Guerci VI, Ciana G, Addobbati R, Grasso D, et al. 2006. Substrate reduction therapy in the infantile form of Tay-Sachs disease. Neurology 66: 278–280.
5. Beutler E, 2006. Gaucher disease: multiple lessons from a single gene disorder. Acta Paediatr Suppl 95: 103–109.
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