A Rare Lysosomal Storage Disease

Archive for November 2009

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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