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Compassionate Use Drug Application Filing | The Addi and Cassi Fund - Niemann Pick Type C

Thursday, December 2, 2021

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 […]