Evaluation of new therapies in Niemann-Pick type C disease
<p>Niemann Pick type C (NPC) disease is a rare autosomal recessive neurodegenerative lysosomal storage disease caused by a mutation in the <em>NPC1</em> or <em>NPC2</em> genes. The functions of the proteins these genes encode are not fully understood, but are thought to...
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| Format: | Thesis |
| Language: | English |
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2014
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