Substrate reduction therapy in the infantile form of Tay-Sachs disease.
Substrate reduction therapy (SRT) with miglustat has been proposed for treatment of some lysosomal storage disorders. Based on the positive experience in Gaucher disease and experimental data in Tay-Sachs (TSD) and Sandhoff animal models, the authors investigated the clinical efficacy of SRT in two...
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Bibliographic Details
| Main Authors: |
Bembi, B,
Marchetti, F,
Guerci, V,
Ciana, G,
Addobbati, R,
Grasso, D,
Barone, R,
Cariati, R,
Fernandez-Guillen, L,
Butters, T,
Pittis, MG |
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| Format: | Journal article
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| Language: | English |
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| Published: |
2006
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