What is Niemann-Pick Type C
Impact of Niemann-Pick Type C
Actelion Pharmaceuticals LTD produced a short video on two families and how Niemann-Pick Type C Disease has impacted their lives. Hollie Carter and Annie Pyne are featured in this video. They both are youngsters living with this disease. At first sight we all instantly start to evaluate what someone with Niemann-Pick Type C looks like or any other debilitating disease. The important feature to note is this disease can strike at any time. Some look completely “OK” and others you can tell something just isn’t right by their actions. Either way this horrible disease claims their life in it’s unrelenting attack on the nervous system.
About Niemann-Pick Childrens Fund
About Us
Niemann-Pick Children’s Fund, Inc. was organized in December of 2008 in order to raise awareness of Niemann-Pick Disease Type C and its affect on families in America; to raise money to promote research to find treatments or a cure for Niemann-Pick Disease Type C: and to provide support to individuals and families affected by this disease through existing channels.
Michael and Jennifer Stults who’s two eldest son’s Brisan and Parker were diagnosed with this rare neurodegenerative disease in August/September 2008 after years of aggravating doctor’s visits trying to understand what is wrong with their children, organized the Niemann-Pick Children’s Fund, Inc. Continue Reading
Blog
20th Annual Family & Medical Conference
This year is the National Niemann-Pick Disease Foundation’s 20th year of providing services to families affected by Niemann-Pick Disease, raising awareness, and the crucial part of funding for research.
This special occasion will be held in Nashville, Tennessee on August 16-19th! More details will develop here in the upcoming future on “the skinny”. Continue Reading
Rare Disease Day 2012
February 29, 2012 is National Rare Disease Day. Even on leap year, we can make this day a special day! What isn’t so RARE about rare disease is the fact that it affects all of us. Although that sounds like a vague statement, let’s think about this a bit more.
A rare disease affects less than 1 in 2,000 people. Chances are, you know someone right now and you might not see them. There are more than 6,000 rare diseases. These loved ones, neighbors, strangers, and people on the opposite side of the earth face a different problem because they have challenges unrelenting. Sadly, they face the same problems. In total, there are hundres of millions of people around the world that are affected by one of those 6,000 rare diseases. Continue Reading
2012 Hospital Hill Run (discount)
The 39th Annual Hospital Hill Run presented by Price Chopper is taking place on June 2, 2012 and the Niemann-Pick Children’s Fund has a great opportunity to help promote awareness of Rare Disease!
We are looking for participants for the UMKC School of Medicine 5k Run\ Walk, 10k Run\ Walk, or the Half Marathon for the NPCF. As a courtesy to those who will Run or Walk, we have the ability to offer 20% off your Race Day entry fee!
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NIH Clinical Trial of Cyclodextrin Update
TRND (Therapies for Rare and Neglected Diseases) and the NIH team met with FDA representatives on December 13th to discuss the progressive plans to bring Cyclodextrin to a clinical trial in 2012. For TRND, Niemann-Pick Type C is one of six pilot projects that were selected.
The NNPDF does a wonderful job of keeping families and people interested up to date on how things are progressing in the disease community for Niemann-Pick Type C. You can visit updates on their page here: http://www.nnpdf.org/aboutus_14.html Continue Reading
