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

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 […]