In its July 24, 2010 edition (click here for full article), THE WALL STREET JOURNAL reported that NPC disease has been selected as one of only three diseases initially selected for a new program at the National Institutes of Health called Therapeutics for Rare and Neglected Diseases or TRND. Due to the initial success of …
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Niemann-Pick Type C News
Research Update – June 2010
DART and 4 other NPC family foundations continue their support of the SOAR-NPC collaborative effort. SOAR-NPC is in essence a virtual biotech, pharmaceutical enterprise seeking a drug cocktail to arrest the progression of NPC. In September 2009, at the National Institutes of Health, SOAR-NPC’s team was able to initiate its first human trial, and only …
FDA rejects Zavesca panel recommendation.
Unfortunately, in mid-March the FDA advised pharmaceutical company Actelion, which markets Zavesca that they would not yet approve Zavesca for NPC; that more definitive proof is required by the law. We are hopeful that further study efforts will lead to future approval. The consequence of the FDA’s decision is that too many children with NPC …