Monday, August 19, 2019

The Road to Building a New Orphan Drug – Am I Mad?

February 4, 2010 by  
Filed under Cyclodextrin

On February 25-26, 2010, I will be attending the inaugural FDA Build-an-Orphan-Drug-Workshop hosted by the Center for Rare Disease Therapies at Keck Graduate Institute (KGI) in Claremont, Calif.  FDA agency experts will be on hand to provide guidance on applying for orphan drug designation. I will be attending the workshop with Dr. Ron Browne, hired [...]

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Nothing Bundt Cakes for Nothing Bundt Beautiful Birthday Girls

January 26, 2010 by  
Filed under Featured Stories

Addi and Cassi turned six years old on Saturday. Over two years ago when the twins were diagnosed with Niemann Pick Type C disease, I was not sure they would still be with us today. Our little angels are amazing fighters and doing very well on their cyclodextrin infusions. Here is the ice cream cone [...]

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FDA Investigational New Drug Application Documents for Cyclodextrin Treatment for Niemann Pick Type C Disease

September 13, 2009 by  
Filed under FDA Cyclodextrin Filings

Here you will find detailed documents outlining Addi and Cassi’s cyclodextrin infusion treatments. We filed a compassionate use IND application to the US FDA in early 2009 to give intravenous cyclodextrin treatment which was approved.  In September 2010, FDA approved a new request to put HPBCD directly into the brains of the twins.  Addi and [...]

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Extra TV Renegade Doctors Show Covers FDA Approval of Addi and Cassi’s Cyclodextrin Infusions

June 12, 2009 by  
Filed under Videos

Renegade Doctors (Extra) Story about Addi & Cassi Hempel’s Infusions of Cyclodextrin from Addi & Cassi Hempel on Vimeo. Twins Addison and Cassidy Hempel are fighting a disease that steals their memory, but they’re only five years old. Now their mother Chris is on a crusade to stop Niemann-Pick Disease Type C, a disease similar [...]

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The Wall Street Journal Reports On FDA Approval of Addi and Cassi’s Cyclodextrin Treatment

June 5, 2009 by  
Filed under News

By AMY DOCKSER MARCUS April 3, 2009 A Mom Brokers Treatment for Her Twins’ Fatal Illness Bucking Scientific Convention, Ms. Hempel Gets Researchers From Different Fields to Share Data on Potential Therapy From the moment her twin daughters, Addison and Cassidy, were diagnosed with a fatal genetic disease in October 2007, Chris Hempel has been [...]

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