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October 2017 Newsletter

What a great kick off for the “Vision of Hope Campaign”. We are encouraged by how many of you have joined our vision for a stronger community, united by our struggles, passions and common goals.

There are many reasons to support the “Vision of Hope Campaign” and here are just a few…

Over the past 25 years the NNPDF has endured with a mission to support families and keep our community connected. NNPDF provides essential, unbiased information to manage the challenging and difficult journeys of families facing Niemann-Pick Disease. 

NNPDF has promoted research in order to fight back and bring greater awareness of the disease. NNPDF has been a leader in making grants to the next generation of investigators who serve as fellows in leading academic institutions. NNPDF has also developed key relationships with current industry partners. We are also committed to being a partner with future ones interested in investing in our community.

NNPDF is committed to being that support wherever families are in their journey. It might be as a first point of contact after the devastating news of diagnosis or when families are seeking clinical trial information. It could also be when families are grieving and need to feel the comfort from others who truly understand.

NNPDF is a partner with INPDA and its initiatives, which include the international patient registry. We will have more about our role in this effort over the coming months.

There are many ways to be a part of this movement to come together in support and collaboration with one another. Please visit to learn more about the opportunities available.

Below we’d like to extend a thank you to those families
who are stepping forward in various ways to fundraise
and support the NNPDF’s “Vision of Hope Campaign”:

Dillon’s Army Fundraisers (100% to NNPDF)

Golf4Garrett Fundraiser (100% to NNPDF)

Walk For Kailey (100% to NNPDF)

Donations for Anne O’Connor Smith’s Birthday (100% to NNPDF)

Mike Smith Triathlon in Honor of Anne O’Connor (100% to NNPDF)

Turley Tot Collection (only) Soy Candles (100% to NNPDF)

Yankee Candle Fundraiser until 12/31/17 (40% of sales to NNPDF)
Group# 99832018

Thanks $4 Giving (1/3rd of total donations to NNPDF)

Jamberry/JamBeauty Fundraiser (at least 25% of sales to NNPDF)

No Pucker Challenge (5% to NNPDF)

Crab Cakes for Caleb (percentage to NNPDF)

Other Fun Events Taking Place This Month

Bake Sales

Hat Day at School

Facebook Fundraiser Link

Restaurant Patron Donations

Dining for Dollars
A restaurant has a special night for a cause and funds are donated. 

Wine Tasting Parties


We are all in this together. Contributions from each of you will lead to a supportive and committed community that continues to gain the interest of researcher and industry partners.

If you haven’t already participated in the ‘Vision of Hope Campaign’ please consider how you, your family or organization might be able to contribute. Only together, can we move towards a hopeful future!

We are so grateful for all of the support from individuals and from those families with fundraisers on behalf of the community and NNPDF “Vision of Hope Campaign”.  

Blessings to you and your families,

Lisa Chavez
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!

Families Helping Families is a support program that was recently developed to offer NPD families a chance to help other NPD families with items that might make their lives a bit easier. These items can be in the form equipment, clothing, diapers, formula, medical supplies, etc. We welcome any ideas, tips or tricks you have found helpful as well.

The NNPDF Equipment Exchange is a part of the Families Helping Families program. As NPD patient and family needs change, equipment may become available that could be used by another family. The NNPDF 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.


Convaid – Rodeo Tilt Pediatric Stroller

Recently available is this Convaid Rodeo Tilt Pediatric Stroller transit model w/all attachments for bus transport, etc. Also includes a tray. Seat width is 14″, weight capacity is 100 lbs. Contact the NNPDF for additional information.

Do You Care for Someone Rare?

To study the impact of rare disease on unpaid friends and family members who provide care (known as “family caregivers”), the National Alliance for Caregiving and Global Genes, launched a “first-of-its-kind” national snapshot of rare disease caregivers. We are collecting feedback from over 1,000 family caregivers of children and adults with rare diseases.

We need your help! We are asking each Foundation Alliance member to ask at least five caregivers from your community to complete the survey.

This research study will be used to tell members of the U.S. Congress, federal agencies, health care providers, health care insurers, researchers, and others about the needs of those who are caring for a person with a rare disease.

YOU can share your experience through a free, anonymous online survey, which will take about 15 minutes to complete. Your information will not be shared or sold, but you can sign UP to participate in future research.

Your experience will help make the case that people who care for someone with a rare disease need support in the workplace, from health care providers, from social networks, and others. Findings will be shared at national and international meetings to help identify solutions for rare disease caregivers.

Thank you for your help!

Always in Hope,
Global Genes

Clinical trials are currently underway studying treatments for Niemann-Pick Disease. For the latest Niemann-Pick Disease research updates, follow the links below.

Please note that patient liaison information has been added to the web page for each pharmaceutical company.


Sanofi Genzyme Update
on ASCEND Clinical Trial Sites


Clinical Trial Update #17


CTD Announces Approval of Application to
Conduct Phase I/II Clinical Study of
Trappsol(R) Cyclo(TM) in NPC Patients in Israel

CTD Enrolls First Patient in US Phase I Clinical Trial
of Trappsol(R) Cyclo(TM) for Treatment of NPC


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.


For the most current NPD information sign up to receive our blog update notice via email. This is a free service provided to our NPD community and signing up is easy! Just click the button below.

9 8.16

PO Box 49, Fort Atkinson, WI 53538-0049
Phone: 920-563-0930  |  Toll Free: 877-287-3672 |


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