February 2018 newsletter
Dear NPD Family and Friends,
All of us have experienced the questioning look when we share the diagnosis of Niemann-Pick Disease, and those affected by any rare disease often get it too. February 28th is a day set aside to recognize those diagnosed with a rare disease. This is a great opportunity to join our voices with other rare disease communities and raise awareness. Media will be looking for stories related to rare disease on this day. We encourage you to share your story with your local news outlets or on social media. Every story shared is a voice for our community and an opportunity for rare diseases to be recognized and supported.
Recently, Justin Hopkin, NNPDF Vice Chair, was able to represent our community at the 14th Annual WORLD Symposium (We’re Organizing Research on Lysosomal Diseases). He was updated on current lysosomal research, met face to face with our industry partners and discussed the next steps for the INPDR (International Niemann-Pick Disease Registry) with our international partners.
On your behalf, we are discussing with each of our industry partners the importance of having information throughout the clinical process and how vital that information is to the decision making of families. This includes the development of the NNPDF Industry Policy Statement and the creation of a system that would allow families to give feedback to industry. We will be sharing the policy and more of that process with you once it is fully developed. Related to this subject is an article from the Orphanet Journal of Rare Disease. This article speaks to the principles for interaction with biopharmaceutical companies and is in line with the goals and policies that the NNPDF is establishing. Click here for the complete article titled Principles for interactions with biopharmaceutical companies: the development of guidelines for patient advocacy organizations in the field of rare diseases.
As the U.S. Representative for the INPDR, the NNPDF remains committed to working with the team leaders to determine the best process for engaging and promoting both patient and clinician data entry into the INPDR here in the United States. We will provide information on that process as the team defines our role moving forward.
We continue to receive positive feedback for the direction our community has taken from our stakeholders throughout the world. They are impressed with the progress and unity we are demonstrating and are eager to support our next steps.
As way of update, the Executive Director Search Committee is diligently working to recruit a leader for the community. Click here to learn more.
In closing, don’t forget to SAVE THE DATE! This year’s 26th Annual Family Conference will be held August 2-5, in Louisville, Kentucky. This is a great opportunity to meet families who share many of the same feelings and experiences you do throughout the NPD journey. At this conference you can obtain vital information to clinical trials, learn of current research and gain the emotional support we all need to keep our hope alive until NPD is defeated!
Blessings to you and your family,
NNPDF Board Chair
DONATE NOW to the Vision of Hope Campaign through PayPal using your debit or credit card. Using PayPal is easy and having an account is not required.
If you prefer to donate via check please mail your gift to:
NNPDF | P.O. Box 49 | Fort Atkinson, WI 53538-0049
Make checks payable to:
National Niemann-Pick Disease Foundation or NNPDF
Just a reminder to mark your calendars for the 26th Annual NNPDF Family Conference to be held in Louisville, Kentucky from August 2nd to August 5th. Plans are underway and we look forward to giving you more details in the Spring of 2018.
Visit gotolouisville.com/things-to-do/ to check out all there is to do in Louisville and start making your plans.
Show your rare. Show you care.
Rare Disease Day® is February 28th. To get involved in Rare Disease Day awareness visit rarediseaseday.org. U.S. participants can find helpful resources, activities and events to get involved at rarediseaseday.us.
NNPDF Rare Disease Day Participation
To raise awareness for Niemann-Pick Disease and Rare Disease Day on February 28th, the NNPDF is offering customized Facebook profile pictures.
Please email your request to Jill at email@example.com.
In your request be sure to include:
- A photo of your NPD loved one
- In Honor of or In Memory of
- Your loved ones name
- Niemann-Pick Disease type
Along with your custom Facebook profile picture, our 2018 World Rare Disease Day Facebook banner will be included for you to display to promote awareness.
The ESPN Infiniti Coaches Charity Challenge is going strong. This year, take a daily TIMEOUT to vote for Matt Painter from Purdue University. Vote daily to help win $100,000 for Niemann-Pick Disease research.
1. Go to espn.com/Infiniti
2. TWEET or RETWEET a TIMEOUT photo with the hashtags #Timeout2Vote #CoachMattPainter #contest
As NPD patient and family needs change, equipment may become available that could be used by another family. The NNPDF Equipment Exchange program assists families who wish to donate this equipment to help others in need. The foundation posts descriptions of equipment available and facilitate any equipment requests. Click the special equipment button for updates on equipment availability.
NEW ITEM AVAILABLE:
We have 2 RespirTech – ClearChest model compression vests available. Comfort band style, size medium with adjustable band. Family states one is a smaller version. For more information contact the NNPDF central office at firstname.lastname@example.org.
Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to support NPD fundraisers.
Awareness Events promote awareness to the general public about Niemann-Pick Disease.
Click the Fundraising button to view current and past NPD events.
These are the updates the NNPDF has received since the last newsletter with regards to clinical trials. As a reminder, new information is immediately posted to the website as soon as it is received. Follow this link: nnpdf.org/research/
Please review our Clinical Trial Update procedure for alerting you to these updates as they are made:
1. The office posts information received about trials and expanded access therapies from companies on the NNPDF website under the appropriate pharmaceutical company.
2. When information is received, it is posted to the website and a summary post is made on the NNPDF Facebook Page and/or NNPDF Awareness Group, with a link back to our website where you can find more detailed information.
3. All updates from pharmaceutical companies are included in the next monthly e-newsletter with links back to the original posting on the website.
A quick reminder that if you are not receiving our NNPDF monthly newsletter via email or have recently changed your address or email, please click the button below to join us or to update your information.