Message from the Executive Director

News & Media

Dear Friends and Families

October Awareness was a flurry of activity. We had video posts, a day of giving, the new INPDA Newsletter, International Niemann-Pick Awareness Day, daily Quick Fact posts, awareness-oriented social media profile pictures frames, and more, throughout the month.

This month we launched our new Pyramid of Support campaign geared towards showing community pride in supporting NNPDF. Support comes in all forms—through volunteerism, through sharing NNPDF news with those within the NPD community and outside in the general community, through raising awareness of NNPDF through fundraisers, and through donations. Support comes from helping one another. I love the metaphor of “Support” for NNPDF and support for one another, and also the symbolism of the pyramid holding each other up and physically supporting one another.

Our ability to raise awareness of NNPDF and Niemann-Pick disease doesn’t end after October. Over the coming months you will see a series of photos and videos made by community members displaying their Pyramid of Support. Please join us—make a pyramid with your friends, family, co-workers, kids’ sport teams or classes, or with anyone you choose, and tag it with #pyramidofsupportnnpdf and share on social media. Help us keep raising the level of Niemann-Pick awareness.

One way that we do this as a national patient organization is through our role in advocacy at the national level. This month, NNPDF collected family impact statements from families throughout the US who wanted to share personal observations of the impact of adrabetadex, an experimental therapy, on their loved ones with the US Food & Drug Administration (FDA). We advocate for the community by participating in important community forums like the Patient-Focused Drug Development meeting held in March which highlighted patients’ and families’ general experiences with Niemann-Pick Type C without focusing on any specific experimental therapies. We also advocate for the community by reaching out directly to the FDA as a conduit for the Niemann-Pick voice. The family impact statements sent to the FDA this month were a response by NNPDF to many NPC community members’ desire to have their voice heard in the regulatory process and to advocate positively for their loved ones about their observations from this specific experimental therapy. We are pleased to share that this information was received by the FDA and to convey their sincere thanks to the patients and families for putting the document together.

As your national patient organization, we will continue to advocate with you and on your behalf for access to all treatment options, to help remove any barriers to these treatment options in the future, to elevate the voice of Niemann-Pick families, and to continue to raise awareness of this devastating disease. Raising awareness of Niemann-Pick disease doesn’t stop after October. Please join us in our ongoing efforts to make our voices heard.




Joslyn Crowe, MSW, MA
NNPDF Executive Director

Supporting one another. Supporting our community.

Taking care of you in challenging times

News & Media

Managing self care forum

Dear Friends,

Are you searching for manageable ways to take some time to care for yourself especially during moments of stress and uncertainty?

Please join NNPDF and NTSAD as we jointly present the upcoming forum Taking Care of You: Self-Care in Challenging Times led by Shoshanna Hecht, LCSW, on Wednesday, April 1, 2020 at 2:00 pm EST/11:00 am PST via Zoom Webinar.

This community forum will include practical tips on manageable, every day self-care, a guided meditation, and time for Q&A.

Shoshanna Hecht is an Executive and Personal Coach based in New York City, with previous experience of over 10 years as a clinical therapist on the west coast. Shoshanna also leads guided meditations to boost confidence while reducing stress, self-doubt, and overwhelm. They can be found both on her website and on the Meditation Studio App named one of Apple’s 10 Best Apps of the Year.

Supporting one another. Supporting our community.

Supporting One Another

News & Media

Keeping our families healthy is always a top priority. COVID19 has made this tougher than ever.

We know that many of our families are facing challenges right now and NNPDF is here for you – we are here to help, to talk, to help find resources for your family, or just to listen. 

We’ve added more Family Services programs with more ways to connect, share, and learn for families at all stages of the Niemann-Pick journey, and we’re helping community members through our Emergency Hardship Program. 

NNPDF is grateful to all our supporters for their gifts. Your support is needed now more than ever. If you’d like to help us to continue to serve the needs of our families, we appreciate your generosity and commitment.

Supporting one another. Supporting our community.