Substrate reduction therapy for lysosomal storage diseases

Author:

Cox T

Publisher

Wiley

Subject

General Medicine,Pediatrics, Perinatology, and Child Health

Reference24 articles.

1. Conzelmann, E and Sandhoff, K. (1983) Partial enzyme deficiencies: residual activities and the development of neurological disorders Dev Neurosci, 6, pp. 58 - 71.

2. Vunnam, R and Radin, N. (1980) Analogs of ceramide that inhibit glucocerebroside synthetase in mouse brain Chem Phys Lipids, 26, pp. 265 - 78.

3. Inokuchi, J and Radin, N. (1987) Preparation of the active isomer of 1-phenyl-2-decanoylamino-3-morpholino-1-propanol, inhibitor of murine glucocerebroside synthetase J Lipid Res, 28, pp. 565 - 71.

4. Lachmann, R. (2003) Miglustat. Oxford GlycoSciences/Actelion Curr Opin Investig Drugs, 4, pp. 472 - 9.

5. Shayman, J and Abe, A and Lee, L and Wild, S and Hiraoki, M. (2002) Small molecule inhibitors of glucosylceramide synthase for the treatment of Gaucher disease Clinical Perspectives on Lysosomal Storage Disorders, 10, pp. 5 - 13.

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