National Niemann-Pick Disease Foundation Mission & Goals
The National Niemann-Pick Disease Foundation, Inc. (NNPDF) is a non-profit, patient advocacy and family support organization dedicated to supporting and empowering patients and families affected by Niemann-Pick disease, through education, collaboration and research. Founded in 1993, NNPDF serves families throughout the nation at all stages of their Niemann-Pick journey. The NNPDF is the US member organization of the International Niemann-Pick disease Alliance (INPDA).
Vision for the Future
We envision an enduring patient support community that embodies strength, hope and empathy for those affected by all Niemann-Pick disease types. We believe deeply in the future of research that promotes clinical treatments with potential to improve patient quality of life and with intent to ultimately cure all Niemann-Pick disease types.
We are committed to:
- Supporting and empowering patients and families affected by NPD by meeting them where they are in their NPD journey.
- Upholding a balanced, unbiased approach with families, researchers, clinicians and industry to move treatment forward to provide a variety of therapies and to work together toward a cure. We encourage full transparency among all stakeholders in the Niemann-Pick disease community.
- Supporting basic science and clinical research through awareness and collaborative funding initiatives.
- Adhering to a written code of conduct that fosters respect for all, regardless of treatment choices or research perspectives, which maintains patient privacy and follows the national HIPPA regulations.
- Encouraging and promoting advocacy and collaborative community partnerships across the NPD community, both in the U.S. and internationally.
- Respecting the diversity of Board members, employees, families, researchers, clinicians and industry, and governing the NNPDF ethically and transparently according to NNPDF Bylaws.
The National Niemann-Pick Disease Foundation is a national non-profit 501(c)(3), rare disease network of families dedicated to providing empowerment resources, support and education for families, caregivers and patients affected by all Niemann-Pick disease types.
We are families, patients and compassionate friends across the U.S. that share hope, promote research and raise awareness for NPD. We are dedicated to supporting and empowering one another, through education of potential therapies for all Niemann-Pick disease types. We work tirelessly as volunteers, caregivers, counselors, and fundraisers for patient support, research and clinical treatment.
On February 10, 1992, six families with children suffering from Niemann-Pick disease met at the National Institutes of Health at Bethesda, Maryland. Concerned with cutbacks in funds for medical research and knowing without research there was little hope for their children, they decided to form a foundation – The Niemann-Pick Type C Foundation, Inc. Bylaws were drafted and a board of directors was selected. Service activities began immediately and the first year saw many accomplishments.
- A Newsletter offering information and support was sent out.
- A Directory of families affected by NPC was compiled and distributed to the families.
- An informational pamphlet was written and mailed to hundreds of professionals and organizations in the medical field to create awareness of this rare disease.
- Fundraising began to channel money into medical research.
- A Scientific Advisory Board of doctors and researchers was established.
- A paragraph was added to the Appropriations Bill for NIH to ensure continued funding for Niemann-Pick disease research at the federal level.
1993 was another busy year. The Foundation name was changed to the National Niemann-Pick Disease Foundation, Inc., nonprofit status was awarded by the IRS, the first family conference was held, and the first research grant was awarded to Dr. Eugene Carstea to identify the genetic cause of NPC. Dr. Carstea announced the discovery of the NPC1 gene four years later.
All of these services have continued and expanded over the years.
- The enewsletter goes out to over 1000 households.
- The NNPDF is a co-founder of the International Niemann-Pick Disease Alliance (INPDA)
- The Family Conference has grown 15-fold with separate sessions for ASMD and NPC. Top researchers attend to interact with families and share their expertise.
- NNPDF was a founding member of the Lysosomal Disease Research Consortium, which awards over $1,000,000 in related research grants annually.
The National Niemann-Pick Disease Foundation currently has over 450 Niemann-Pick families as members. Thousands of medical, corporate, professional, friend and family sponsors assist in supporting our goals through contributions and donations of services. NNPDF is the largest Niemann-Pick disease family support organization in the world and one of the largest private funders of Niemann-Pick disease research.
The NNPDF’s board continues to be comprised mainly of parents with children affected by NPD. All board members donate their time to the NNPDF on a volunteer basis.
Of the six original families, all have lost a child to Niemann-Pick disease. All six are still NNPDF members.
The foundation has a record of getting things done. The NNPDF was founded in 1992. Since then, the NNPDF has:
- Raised over $5.5 million for research on NPA, NPB, and NPC, family support, education, and other activities
- Funded research that led to the discovery of the NPC1 gene
- Funded research that has led to the identification of numerous gene mutations for use by researchers, clinicians, and in genetic testing
- Funded research to study the neurological effects of NPD
- Co-sponsored the first International Symposium on NPC
- Promoted awareness of NPD by publishing educational materials
- Held an annual conference every year since 1993 for families affected by all types of NPD, scientists, and others
- Published regular newsletters to keep families informed about the Foundation and the latest advancements in understanding NPD
- Provided opportunities for NPD families to communicate with each other and share their thoughts, ideas, and experiences