November 2017 Newsletter
Dear Family and Friends,
We are very encouraged by the community support for the Vision of Hope Campaign. In August, at our family conference, we shared with you the need to strengthen and unify our community with one patient voice. We explained that to do this, we need a trained non-profit leader, who is skilled at hearing your individual voices and weaving them together to create one strong community voice; someone who is able to effectively communicate and leverage opportunities that will amplify that voice as we work towards our shared goals. We also shared the financial commitment needed that will allow us to provide some of the services you identified and to recruit a new leader.
Our community goal for Vision of Hope is $150,000.00 and we are pleased to report that over 300 of you have already taken time to evaluate how you can help. Through individual gifts, fundraising events and promotional efforts you have brought us half way to our goal. As fundraisers wrap-up and end of year gifts are made, we are confident we can reach our goal and begin our search for a trained non-profit leader who can manage the complexities of today – a leader supporting research efforts, managing industry relationships and helping us to shape a unified voice.
The Reinvention Advisory Committee (RAC) is supporting and guiding the board through the search process for an executive director. We will continue to update you as we move forward. As a reminder the RAC is made up of Lisa Chavez, Dr. Andy Lieberman, Dr. Marc Patterson, Dr. Ed Schuchman, Dr. Justin Hopkin, Jill Flinton, Jamie Ring, Barbara Wedehase, Michelle Davis and Amy Kant. Jamie Ring brings many years of management experience in rare disease non-profits and industry, including her former role leading Genzyme global patient advocacy. As well, both Barbara Wedehase, former Executive Director of the MPS Society and Michelle Davis, current Executive Director of the IFOPA, bring years of leadership experience in the rare disease community and in building communities. We are delighted that they can share their expertise to help us reach our goals for a new Vision of Hope. For more information about the Reinvention Advisory Committee members please go to: nnpdf.org/reinvention-advisory-committee/
Thank you to each of you who have contributed to our Vision of Hope campaign. If you have not yet had an opportunity to support Vision of Hope, please consider how you might be able to help us toward our goal of working together for a stronger community with renewed strength and determination to cure NPD. Within this newsletter are some great gift giving ideas that benefit the foundation.
Please also take time to review the NPD Registry Update from Justin Hopkin, NNPDF Vice-Chair.
With blessings to you and your families, we wish you a warm Thanksgiving Holiday.
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. Click the button to the right!
I am sharing with you an update from the International Niemann Pick Disease Alliance meeting that Lisa Chavez and I attended in Toronto from October 13-15. Overall the meeting was very positive citing the progress the community has made in the last few years with three NPC trials and 2 ASMD/B (adult & pediatric) trials either completing enrollment or still enrolling patients.
We received very positive feedback from the international community about our family conference in Chicago. The importance our community placed on having a unified patient voice from an unbiased source resounded with our international partners.
Significant progress has been made on the patient registry, which is NOW AVAILABLE for enrollment. Please take the time to enroll at INPDR.org. I completed the process from start to finish in about 20 minutes. The only supporting documentation requested (not required) for the patient registry is genetic test results. If those are available, they can be uploaded during registration. Please take the time to complete this. If you have lost a loved one, we would appreciate having their information placed in the registry as well. This registry will be an asset to the community as a useful tool for clinicians and researchers in the future.
The second part of the registry is for health care professionals to input clinical data. Please encourage your provider to complete this portion of the registry. Any questions about the registry can be directed directly to the INPDR on the website. Please let the NNPDF office know if you have unresolved concerns with the registry that we can share with our partners.
Looking for holiday gifts for others or even a gift for yourself? Check out the NNPDF Store for great items for family and friends. Your purchase not only helps support the NNPDF but also raises awareness of Niemann-Pick Disease. Visit nnpdf.org/store/ to place your order today!
Another easy way to help support the NNPDF while you are shopping online is by using AmazonSmile for all of your Amazon purchases. You shop, they give. AmazonSmile is a simple and automatic way for you to support the NNPDF every time you shop with 0.5% of your purchase price donated to the NNPDF. Click here to register today!
Check out the NNPDF Shop and Support page for additional ways you can shop and support the NNPDF online.
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:
- The NNPDF website is updated with the new information under the specific pharmaceutical company.
- An update is posted on the NNPDF Awareness Facebook Page linking individuals back to said page on the NNPDF website.
- The update is then saved to be put in the next enewsletter that comes out via email about the third Tuesday of each month.
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