Hello NNPDF Families and Friends,
We have received an additional update from the Therapeutics for Rare and Neglected Diseases ~ Niemann-Pick Disease Type C Team (TRND NPC Team) at the National Institutes of Health (NIH) with new information on the Phase I Study of the Cyclodextrin (HP-b-CD) for NPC1 Disease.
The application for an Investigational New Drug (IND) was filed with the United States Food and Drug Administration (FDA) on Wednesday, November 14th, 2012! Please follow the link below to the NNPDF "Cyclodextrin" web page for more details.
Sincere and heartfelt THANKS to ALL those who have worked to diligently to see this program move forward. We WILL Persevere in our Quest for a Cure!