October 2019 Newsletter
Message from Executive Director
Niemann-Pick Awareness Month
Newborn Screening | NORD ASMD & NPC Video Resources
INPDA Newsletter | Home Runs for Heaven
NNPDF In Action | NNPDF Emergency Hardship Program
Clinical Trial Update | Community News
Fundraising & Awareness Events | Save the Date
Upcoming Events | Stay Connected With Us
.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
Thank you to all those who participated in SUPPORT of the NNPDF and awareness of Niemann-Pick Disease during October Global Niemann-Pick Disease Awareness Month! Many awareness activities were happening throughout the month:
- INPDA Newsletter Inaugural Issue
- NNPDF Giving Day
- Global Niemann-Pick Awareness Day
- NNPDF Pyramid of Support
- Quick Facts
- NNPDF SUPPORT Videos
Raising awareness of Niemann-Pick Disease doesn’t stop for us after October, we will continue to provide education and resources to Niemann-Pick families, physicians, and the general community throughout the year. Check our online resources or contact Laurie Turner, Family Services Manager, for a discussion at firstname.lastname@example.org.
Send us your Pyramid of Support photos in SUPPORT of the NNPDF and ALL NIEMANN-PICK FAMILIES!
First-Ever NPC Newborn Screening Pilot Study
It’s official! The ScreenPlus program in New York state that will include NPC received a major award from the National Institutes of Health that will allow the program to move forward. ScreenPlus is the most expansive consented pilot newborn screening study in the country, where families of newborns born at 8 New York hospitals will be offered the option to have their babies screened for an additional 13 disorders – including Niemann-Pick C. This is the first time any newborn, anywhere in the world, will be given the option of being screened for NPC at birth.
The Firefly Fund’s NPC Newborn Screening Working Group member Dr. Melissa Wasserstein of Montefiore Hospital in NY is leading the study. And Firefly is one of the funders supporting the program through a unique cost-sharing collaboration that also includes academic centers and several pharmaceutical companies.
NNPDF joins Firefly, all its Working Group members and supporting NPC stakeholders in congratulating Dr. Wasserstein on this major accomplishment. This study is history-in-the-making as the NPC community will begin to gather evidence regarding the importance of early diagnosis and intervention in the treatment of NPC. Learn more by clicking on this official announcement – and share the news with your networks!
The National Organization for Rare Diseases created animated educational resource videos on ASMD and NPC to help raise awareness of Niemann-Pick Disease. Video links can be found on our NNPDF video resources webpage as well as on NORD’s website. Thank you to NORD for your partnership!
In a global collaboration effort, the International Niemann-Pick Disease Alliance (INPDA) published its first newsletter in early October. INPDA President Sandy Cowie writes “the newsletter will serve as a communication tool for all of the members of the INPDA to share information and updates and will be released in an electronic format 3 times per year.” The October issue features updates from 12 INPDA member groups. Click here to view in full.
Home Runs for Heaven is a book written by Gary McCusker and Cindy Kay Zimmermann (mother to Patrick, NPC 2/12/2000 – 9/24/2016). Together they share a very moving account of Patrick’s life story and how their family helped each other through every difficult transition of the Niemann-Pick journey. Cindy shares a great deal of information about how the disease affected Patrick and hopes that their experiences can help other families dealing with Niemann-Pick disease. Click here for more information.
The NNPDF Emergency Hardship program offers help to qualified NNPDF U.S. member families facing a crisis. Funding includes but is not limited to, specialized medical equipment, durable medical goods, utility bills (heating and cooling, electricity, phone, water and sewer), home and car repairs, and bereavement expenses. This assistance program provides relief to members facing a situation that threatens their immediate health and safety, or that causes a terrible hardship. It is intended to provide short term financial assistance in a crisis situation and is not intended to address long term financial challenges.
If you have questions about this program, please contact Laurie Turner, Family Services Manager at 603-413-8707 or email@example.com. Applications will be reviewed by the NNPDF Emergency Hardship Program Committee consisting of 2 Board members and 3 members-at-large.
To be eligible for funding, NPD individuals and families must reside in the US and must be enrolled as a member of the NNPDF for a minimum of 6 months prior to requests*. Funding is available for up to $750 per calendar year per eligible member. Families with multiple children with NPD are eligible to apply for each affected individual. The NNPDF Board of Directors and Emergency Hardship Program Committee members work to keep the fund fiscally sound, by being the last resort to be used, limiting regular, recurring use by any one recipient, and encouraging donations.
*Those with new diagnoses are eligible to apply if diagnosis was within 6 months of request.
Clinical trials are currently in progress to study and develop treatments for ASMD and NPC. The NNPDF posts new information regarding clinical trial updates as soon as it is received. Visit our Clinical Trials web page for up to date information on all clinical trials.
Updates from Cyclo Therapeutics:
Cyclo Therapeutics, Inc. today announced its publication of the most extensive set of case studies to date on expanded access use of hydroxypropyl beta cyclodextrin to treat patients with NPC. Click here for complete announcement.
Cyclo Therapeutics announces completion of enrollment in its Phase I trial to evaluate Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C. Click here for complete announcement.
Cyclo Therapeutics, Inc. announces Maurizio Scarpa, MD as EU Coordinating Investigator for the company’s pivotal trial in Niemann-Pick Disease Type C. Click here for complete announcement.
Updates from Mallinckrodt Pharmaceuticals:
Mallinckrodt shares an update on adrabetadex (VTS-270) to the NPC community. Click here for complete message.
Mallinckrodt resumes its VTS301 study in France. Click here for complete announcement.
Mallinckrodt resumes its VTS301 study in the UK. Click here for complete announcement.
Newborn Screening Historic News: First-Ever NPC Newborn Screening pilot study program moves forward. Click here to read more.
Cyclo Therapeutics announces support for The Firefly Fund’s Newborn Screening Program. Click here to read more.
CTD Holdings Announces Completion of Name Change to Cyclo Therapeutics, Inc. Click here for complete announcement.
INPDA Newsletter launched in early October! Click here to view in full.
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 host and support NPD fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease. Click here to view current and past fundraising and awareness events.
THANK YOU to those of you who have already hosted a Facebook Fundraiser! Are you interested in hosting? It is a great opportunity to raise awareness and funds for the NNPDF. If you are hosting a Facebook fundraiser please email the NNPDF Central Office at firstname.lastname@example.org to let us know..
Yankee Candle Fundraiser
Yankee Candle Online Fundraiser in memory of Bryanna Desouza and in honor of all Niemann-Pick Disease families started July 11th and runs through January 8, 2020. Click here for complete details.
Bryanna Desouza (Jan 20, 1992 – Dec 5, 2006) NPC.
Jamberry Niemann-Pick Disease Nail Wraps
Show your support in the fight against Niemann-Pick Disease with these custom Jamberry nail wraps. 30% of all sales will be donated to the National Niemann-Pick Disease Foundation for family support services. Click here for ordering details. Available year round.
Have you hosted a fundraiser for NNPDF recently?
Send us your photos and we’ll share them and details from your event in upcoming newsletters!
Fundraisers in Memory of Dillon Papier
The Papier’s recently hosted their 13th Annual Dillon Papier Charity Golf Tournament, in addition to their 9th Annual Baltimore Orioles Dillon Papier Benefit. Friends and family attended both events, including some very special NPD families. Photos from the events can be found at Charity Golf event and Baltimore Orioles event.
Save the Date!
NNPDF Family Support & Medical Conference 2020
Mark your calendars for July 9-12, 2020 to join us at the NNPDF Family Support & Medical Conference – including Family Advisory Working Groups – in San Antonio, Texas!
Check out our Upcoming Events page at nnpdf.org/news-media/upcoming-events/.
NNPDF Industry Update Webinar Series
Dates coming soon!
Rare Disease Day
February 29 | Watch for details!
NNPDF Family Support & Medical Conference 2020
July 9-12, 2020 | San Antonio, Texas
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 here to join us or to update your information.