This year is the National Niemann-Pick Disease Foundation’s 20th year of providing services to families affected by Niemann-Pick Disease, raising awareness, and the crucial part of funding for research. This special occasion will be held in Nashville, Tennessee on August 16-19th! More details will develop here in […]
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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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One thing that I’ve personally learned is not to get excited about many things when it comes to research for Niemann-Pick Type C. The job of PR firms and Press Releases are to generate buzz, hype, excitement, and positive feelings. Sometimes there are a lot of finite […]
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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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