A Rare Lysosomal Storage Disease

Archive for October 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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Dementia. It changes your life.

“Dementia is something that is usually associated with older people. However, children, teenagers and young adults can experience dementia as a result of a number of rare diseases and conditions. Niemann-Pick Disease Type C (NPC) is an example of one of these diseases, and one of its main symptoms is dementia.” (Hinton et al, 2005)

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