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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:
Drug Development in Rare Diseases: the Regulatory Path to Approval

Breaking it down: How the NPC-Cyclodextrin process has unfolded
A series of mini-presentations referring to elements of the FDA presentation, including both progress made and challenges confronted

Cyclodextrin therapy for NPC1 disease (Dan Ory)
What this means for NPC patients/parents; one slide that provides a overview of the process and clear places where everyone can see where they fit in (Included in Panel Session Part-I)

Q&A with panel comprised of:

      • Andrew Mulberg
      • Sara Goldkind
      • Denny Porter
      • John McKew
      • Caroline Hastings
      • Patti Dickson
      • Elizabeth McNeil
      • Steven Silber
      • Dan Ory

Click HERE for PART I of the Panel

Click HERE for PART II of the Panel

Closing summary: Dan Ory
(included in Panel Session Part 2)

Acknowledgement of Dr. Mark Kao (J&J) Contributions: Karen Quandt
(included in Panel Session Part 2)

 Click HERE to view the entire Album on VIMEO