Tuesday, March 28, 2017

Dr. Stephen Groft of NIH Visits Global Genes Project In Advance of Rare Disease Day 2011

February 24, 2011 by  
Filed under Featured Stories, Uncategorized

Monday, Feb. 28, is Rare Disease Day 2011.  Here is a great story from the Orange County Register, which is helping to raise awareness for rare diseases and our efforts to spread awareness through our blue denim jeans ribbon and “Wear That You Care” campaign. Local advocates looking to kick-start research to help patients with [...]

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FDA Grants Orphan Drug Status For Cyclodextrin Compound To Treat Fatal Genetic Cholesterol Disease

May 16, 2010 by  
Filed under Featured Stories

Drum roll, please! Dr. Caroline Hastings at Children’s Hospital Oakland and Research Center received a call on Friday afternoon from the U.S. Food and Drug Administration regarding the orphan drug submission we made at the end of February. While we have not received the official letter by mail (probably next week), it appears our Orphan [...]

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FDA Does Not Approve Actelion’s Zavesca in US – Distressing News For Niemann Pick Type C Community

March 9, 2010 by  
Filed under Featured Stories

Actelion, the makers of Zavesca, today put out a brief statement saying that the U.S. Food and Drug Administration did not approve Zavesca and wants more information from the company on treating Niemann Pick Type C, a rare and dementing neurological disease that afflicts our six year old identical twins, Addi and Cassi. Niemann Pick Type C disease is caused [...]

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FDA and EMA Forge Rare Disease Collaboration; Announcement Coincides with World Rare Disease Day 2010

February 28, 2010 by  
Filed under Featured Stories, Health Care Policy

In recognition of World Rare Disease Day 2010, the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) announced that they will collaborate together and now accept a single annual report from sponsors with an orphan drug designation for rare medical conditions. The joint announcement was made by Dr. Timothy Cote, director [...]

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FDA Orphan Drug Workshop Makes Filing Applications Easy – Even For Rare Disease Patient Advocates!

February 27, 2010 by  
Filed under Featured Stories

At a historic orphan drug workshop held by the U.S. Food and Drug Administration Feb. 25-26 and before World Rare Disease Day 2010, we filed a momentous orphan drug application with the FDA for the treatment of Niemann Pick Type C disease with cyclodextrin. Our filing occurred in Claremont, CA, at the Keck Graduate Institute’s Center [...]

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