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Posted by on Oct 4, 2013 in Foundation NewsLine |

October Awareness Month ~ Day 4 ~

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~October 4th, 2013~  
Drug Development in Rare Diseases: The Regulatory Path to Approval
Dear Families & Friends,
On Day 4 of our October NPD Awareness Month event, we are highlighting the Food and Drug Administration’s presentation during the 2013 NNPDF Family Support and Medical Conference, held in Baltimore, Maryland in early August. 
Andrew Mulberg, M.D., representing the FDA, discusses the process to get approval for a rare disease clinical trial. This is especially of interest to the families of the NNPDF as we currently have TWO clinical trials taking place for Niemann-Pick Disease.  
      1. Acid Sphingomyelinase Deficiancy (ASMD) ~ Enzyme Replacement Therapy

                Sponsor:  Genzyme, a Sanofi company, Mount Sinai School of Medicine
                To learn more about this trial ~ follow this link.  
            2.  NIH/TRND Hydroxypropyl-β-cyclodextrin (HP-β-CD) for NPC
                 Sponsor:  National Institutes of Health
                 To learn more about this trial ~ follow this link.

Dr. Andrew Mulberg’s August 2013 presentation and professional information is noted below:

~ October NPD Awareness Month —- Fundraiser Highlight~

Dillon Papier Golf Tournament 

Dillon Papier (NPC) and Family 
This year’s annual Dillon Papier Charity Golf Tournament is scheduled for Friday, October 4th, 2012.  Registration will begin at 9:00 a.m., with a shotgun start at 10:30.  
This is a 4-person scramble format tournament, with prizes for closest to the pin, longest drive, etc., plus raffles, gift bags, and other prize opportunities. 
Visit Upcoming Events for more information.  Intersted in hosting your OWN Golf Outing to benefit the NNPDF ~ check out the NNPDF’s “Recipe for Success” Golf Tournament link here to access the tried and true methods for a successful Golf Tournament. 
~NNPDF October Awareness Month~ 
Every day in the month of October we will be sharing fundraising ideas and highlight special events to keep the momentum going.  So keep an eye on this web site and the Facebook Awareness Event page for the latest updates. 
Remember that any events you are hosting for October can be sent to the NNPDF Central Office to be shared with the NPD Community.  Contact:
See you tomorrow! 
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