A Rare Lysosomal Storage Disease

Archive for 2009

Historic FDA related drug advisory committee will meet | Zavesca

This is an email from Nadine Hill of the NNPDF. This is good news. If the FDA does say lets move forward that means more families might be able to participate because the drug would be FDA approved. All though this isn’t a cure it is one […]

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Today, there is no cure or effective treatment

The NPCF was formed to help create a entity that we could bring to communities that surround us the awareness needed to shed light on a disease that researchers are taking interest in. We felt after facing this reality that research has a different time line than parents yet they are making progress then why could we not help out in some shape, way or form?

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New therapies – Niemann-Pick type C disease

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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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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