Moms Receive EU Orphan Drug Designation From European Medicines Agency For Novel Cholesterol Drug Called Cyclodextrin

Last year, after Dr. Caroline Hastings and I received the U.S. Food and Drug Administration orphan drug designation for Hydroxy-propyl-beta-cyclodextrin (HPBCD) for the treatment of Niemann Pick Type C disease, I was contacted by Sue French. Sue lives in the United Kingdom and her son William is also afflicted with the rare and fatal genetic […]

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

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 […]

How Cyclodextrin Solution Is Made For Addi and Cassi’s Intravenous Cyclodextrin Therapy

A number of people from around the world who have children diagnosed with the fatal cholesterol disease called Niemann Pick Type C  have asked me how our we prepare our cyclodextrin solution for Addi and Cassi’s bi-weekly IV infusions of the compound. All hydroxy propel beta cyclodextrin (HPBCD) is not the same. It’s very important […]

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

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 […]

Anthrax Bacteria Killed By Simple Sugar Compound Called Cyclodextrin. Is CDC Looking Into This?

We all remember the Anthrax-laced letters that killed five people and severely rattled the country post-9/11. Just when you thought there might not be a way to stop this lethal infectious disease along comes beta cyclodextrin, a non toxic sugar compound. A researcher by the name of Vladimir Karginov at a company called Innovative Biologics […]