Global ETD Search
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Showing 1 to 3 of 3 for “"Nephropathic"”.
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Formulation studies on cysteamine for the treatment of nephropathic cystinosis.
Nephropathic cystinosis is a rare autosomal recessive disease characterised by raised lysosomal levels of cystine in the cells of almost all organs. It is treated by regular oral and topical administration of the aminothiol, cysteamine (Cystagon TM), which possesses an offensive taste and smell. …
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The design and synthesis of novel pro-drugs for the treatment of nephropathic cystinosis.
Cystinosis is a metabolic disorder characterised by the abnormal accumulation of the amino acid cystine in cells leading to a slow destruction of all major organs. If patients diagnosed with cystinosis are untreated, death due to kidney failure ensues in the second decade of life. A number of …