Abstract: Niemann-Pick disease type C (NP-C) is an autosomal recessive disorder characterized by progressive neurological deterioration leading to premature death.
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21
Nov
2009
New therapies – Niemann-Pick type C disease
25
Oct
2009
Gene Therapy Halts Brain Disease in Two Boys
By Jocelyn Kaiser ScienceNOW Daily News 5 November 2009 Researchers have used a modified AIDS virus to halt a devastating brain disease in two young boys. The treatment, in which the virus delivered a therapeutic gene, marks the first time gene therapy has been successfully used against […]
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