A Rare Lysosomal Storage Disease

Posts Tagged ‘Therapies’

Promising Therapies for Niemann-Pick Type C Disease

In Rockville, MD on June 3-4th many of the researchers looking into different areas and avenues for Niemann-Pick Type C came together to discuss promising therapies. Although research is making progress, parents of NPC patients never feel that research is happening fast enough.

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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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