Each year October is set aside to have a focal point of Niemann-Pick Disease Awareness Month! The National Niemann Pick Disease Foundation has been promoting this the last 12 years to help create buzz and awareness about this horrible disease. Along with families affected by Niemann-Pick, people […]
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TRND (Therapies for Rare and Neglected Diseases) and the NIH team met with FDA representatives on December 13th to discuss the progressive plans to bring Cyclodextrin to a clinical trial in 2012. For TRND, Niemann-Pick Type C is one of six pilot projects that were selected. The […]
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Karen Quandt is the Board Chair for the NNPDF.org. A few years back she was completing a study about neurodegenerative disease in family members of patients with Niemann-Pick Type C (NPC). There differently is a relationship between NPC and other diseases. At this given time ,the current […]
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This week the FDA has stated formally that they see promise in the use of Cyclodextrin in treating Niemann-Pick Type C (NPC) by granting its “Orphan Drug Designation”. In January of 1983, the Orphan Drug Act (ODA) was passed into law in the United States with consistent […]
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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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