2013 NNPDF Session on Cyclodextrin in NPC Disease
On August 2nd, 2013 in Baltimore MD, the National Niemann-Pick Disease Foundation (NNPDF) hosted a morning session on the use of Cyclodextrin (2-HPBCD) in the treatment of NPC disease during it’s 21st Annual Family Support and Medical Conference.
This session was attended by about 220 people. In many ways, the session was a milestone for the NPC community. This session marked a true coming together of the NPC community along with our partners at the NIH, FDA, J&J, and the numerous foundations that have all contributed to the Cyclodextrin story.
Key messages from the meeting include; 1) The importance of collaboration and cooperation, 2) The need for a clear path forward for NPC patients that cannot participate in the formal studies, and 3) The daunting but surmountable regulatory requirements that make our path forward incredibly challenging.
The following is the agenda. There are links in the agenda to a video from each of the presenters. You can also “browse” the videos using the “next” and “prev” buttons to the right ——–>
2013 NNPDF Session on Cyclodextrin in NPC Disease Welcome: Karen Quandt, NNPDF Chair, and Marc Patterson, moderator Panel: Patient perspectives on Cyclodextrin NPC parents’ experiences: waiting, hoping, parenting, advocating, innovating, etc.
FDA presentation: Breaking it down: How the NPC-Cyclodextrin process has unfolded
Cyclodextrin therapy for NPC1 disease (Dan Ory) Q&A with panel comprised of:
Click HERE for PART I of the Panel Click HERE for PART II of the Panel Closing summary: Dan Ory Acknowledgement of Dr. Mark Kao (J&J) Contributions: Karen Quandt |
Click HERE to view the entire Album on VIMEO |