May 2017 Newsletter
Dear NNPDF Families,
This year we celebrate 25 years of supporting and working with families across the world to create a community that has now attracted the attention of several pharmaceutical companies passionately working to approve a drug for us. It’s amazing what we can do when we work together!
After many months of evaluating the NNPDF’s position within the community, we were so pleased to share with you earlier this month that there is great support and desire for the NNPDF to reinvent our mission in order to focus on family support. Our goal is to provide a central source of information and support for the patient community. This will include providing ongoing family support, unbiased treatment information, education on NPD, updates on the latest research and advocacy initiatives, as well as networking with other organizations both locally and internationally. As a member of the International Niemann-Pick Disease Alliance, we are excited to be chosen as the U.S. coordinator with the INPDA for the launch of the international registry this year. It is critical to have a central patient registry with regard to a rare disease. The start-up of the international registry will likely attract further industry interests. Click here for more information.
The reinvention is a big step and will require the commitment from the whole community to be successful. We are looking at a plan that is realistic and has the needed tools and staffing for a more complex operation that provides an array of services. To do so, we need to restructure our governance including board and membership guidelines, and to develop a community campaign and investment strategy that will enable us to hire an experienced non-profit leader.
We are excited to present our ideas for the future vision at the 25th Annual Family Conference in Rosemont, Illinois from August 10 -13. We hope that you will all join us with matched commitment and willingness to rebuild our community so that we continue to benefit from treatments, while keeping a keen focus on finding a cure for all Niemann-Pick Diseases.
At the conference, we will explore our shared values and develop the principles that we commit to as members. The conference program theme will focus on our future goals as a community, including family services, advocacy, the role of pharmaceutical companies, as well as updates. It will also include a greater focus on breakout sessions that will be designed to support families in the daily challenges that come from living with NPD. You can find the latest conference details and more at nnpdf.org/2017fc.
Looking forward to sharing this hopeful journey with you and spending quality time together at the NNPDF 25th Annual Family Conference.
Blessings to you and your families,
As previously announced the NNPDF will be hosting the 25th Annual Family Conference from August 10-13, 2017 at the Hilton Rosemont/Chicago O’Hare in Rosemont, Illinois.
Within the next week, NNPDF registered members in the U.S. and Canada will be receiving a 2017 Family Conference postcard from the NNPDF providing conference information. You can also visit the 2017 Family Conference web page at nnpdf.org/2017fc for the 2017 Family Conference packet (online only), conference registration, and hotel reservation information. If you have not joined the NNPDF click here to do so.
We are looking forward to celebrating and honoring the 25 years of strength and perseverance that so many have contributed to bringing us to this pivotal moment for our community!
A project initiated by the
“International Niemann-Pick Disease Alliance”
The INPDA and NNPDF are delighted to announce that the NNPDF will be acting as a co-coordinator for information and support for the roll-out of the International registry for NPD in the United States.
It has taken three years and $3m but the NPD world now has a truly international disease registry. This registry has been created by a consortium of NPD scientists, clinicians and patient organiations from around the World. This registry is collecting data from clinicians and affected families.
Unlike most disease registries this registry is owned by the INPDA, a worldwide network of NPD patient support and advocacy foundations, and managed by a team of professionals. The objective of the registry is to facilitate progress by building knowledge about both ASMD and NPC. By understanding the natural history of these diseases it will be possible to evaluate therapies, manage and predict disease progression, recruit for trials and tailor treatments. In brief begin to understand more about these diseases.
The registry is now built and it will be launched in as many countries as possible throughout the World this year. The NPD patient support and advocacy groups will be receiving information about the roll out and will be in touch during 2017.
This initiative has the potential to significantly enhance progress and make a real difference to those affected by Niemann-Pick Disease.
The Niemann-Pick UK group has released a new video on Niemann-Pick Disease Type C. In this video Professor Marc Patterson and Professor Paul Gissen explore the causes, symptoms, and potential therapies for Niemann-Pick Disease Type C. Visit our video resources page for this and other informative videos on Niemann-Pick Disease. Click the button below to view the video titled What is Niemann-Pick Type C?
More than 350 million people worldwide suffer from a rare disease. If a disease affects fewer than 200,000 people in the United States, it is considered rare. There are currently about 7,000 rare diseases identified worldwide, and approximately 80 percent are caused by genetic changes. These diseases are often chronic, progressive, complex, life-threatening, and affect the quality of life.
Global Genes has developed a genetic toolkit titled Genetic Testing: Is This My Path to a Diagnosis? This toolkit is designed to provide rare disease patients and their families an introduction to genetics and genetic testing.
Contributions through fundraising by NNPDF members, families and friends are used to support research toward finding treatments and a cure for NPD. Funds also support the National Niemann-Pick Disease Foundation which provides services to individuals and families affected by Niemann-Pick Disease. Please continue to support the following current and upcoming fundraisers.
Awareness events promote awareness to the general public about Niemann-Pick Disease.
Donating to the NNPDF has just gotten easier. You can now donate online through PayPal using your debit or credit card. Using PayPal is easy and having an account is not required. Click the button to donate now!
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