November-December 2022 Newsletter
Message from the Executive Director | We Remember Them | The Benefits of Having a Great Local Care Team | Dangerous Blues by Stephen Policoff | Gift Ideas Supporting the NNPDF | AmazonSmile | NPC Survey Opportunity | ASMD Accelerate | NPC Sibling Study | Clinical Trial Updates | Community News Updates | Fundraising | Surveys & Studies | Membership | Emergency Hardship Program
Message from the Executive Director and Board Chair
Dear NNPDF Community,
It is hard to believe that we are just weeks away from the end of 2022. And once again this has been a monumental year with many important developments for our community. We are very pleased to have this opportunity to wish you and your family all the best for the New Year and to reflect on some of the important events of 2022.
Heading into 2023, NNPDF will work to expand our efforts and build on the momentum in 2022 in many new ways. We will continue to support and participate in research initiatives and other programs that can help leaders in government, industry, and advocacy better understand the impact of Niemann-Pick disease. We will continue to work with regulators to encourage them to consider the essential new approaches in clinical research that can lead to more new treatments in the years ahead. We also remain committed to fighting for access to treatment for the entire community and will not stop until ALL people affected by ALL types of Niemann-Pick disease have access to life-changing therapies. As the Niemann-Pick landscape changes and treatments do become available, we will also expand support to help families manage issues related to insurance, working with their providers, and life planning.
End-of-year is also a great time to recognize the many examples of strength and determination from our community and thank all the people – healthcare leaders, patients, families, researchers, donors, and industry partners – who supported our work at NNPDF. As we look back on the year, we are so encouraged by the amazing commitment of so many members of our community. We might be small, but we are mighty. The last year has shown us what is possible when we stay strong and work together.
Thank you again for your continued support. We are confident that in 2023 we will break many more barriers and reach new levels of change for the community. The battle is not over, but there is more hope on the horizon than ever before.
From all of us at NNPDF, have a safe and happy holiday.
Joslyn Crowe, NNPDF Executive Director
Becky McGuire, NNPDF Board Chair
The Benefits of Having a Great Local Care
Most patients and families in the Niemann-Pick community are too familiar with the need to travel to access the specialized medical care they need. Among our many services at NNPDF, patients often turn to us to find hospitals and doctors that have specific experience in treating Niemann-Pick disorders… Check out our latest blog post entitled The Benefits of Having a Great Local Care Team.
Dangerous Blues by Stephen Policoff
Congratulations to Stephen Policoff, Niemann-Pick community member, on his recently published book Dangerous Blues. Stephen has generously cited NNPDF as the organization to which he wishes to donate a percentage of the royalties. Steven’s daughter Anna lost her battle in 2015 of NPC-related causes. This novel is dedicated to Anna and to his wife Kate, who also passed away in 2012.
Gift Ideas Supporting the NNPDF
Looking for a gift idea? Check out our Books and Publications webpage. Here you’ll find books authored by Niemann-Pick community members who have expressed themselves in books or other publications. Also included are books by authors that help raise awareness of Niemann-Pick Disease.
Shop AmazonSmile for the Holidays
When you shop AmazonSmile this holiday season, be sure to designate National Niemann-Pick Disease Foundation as your favorite charity! It’s an easy way to make an impact for Niemann-Pick families. Shop AmazonSmile and Amazon will donate to the NNPDF. It’s easy! Learn more and sign up. You shop. Amazon gives.
NPC Survey Opportunity
Quality of life experience in patients with Niemann-Pick disease Type C during the COVID-19 pandemic
Are you an adult who has been diagnosed with Niemann-Pick disease type C (NPC) or the parent/caregiver of someone who has been diagnosed with NPC? If so, we would like to invite you to take part in our research study looking at quality of life in those with NPC. Learn more about this study.
Research reminder from Wylder Nation and NNPDF
Wylder Nation and NNPDF are still enrolling for ASMD Accelerate! Every child’s story can make a difference for the future of ASMD care.
This study is an opportunity to contribute to ASMD research from home. Signing up with PicnicHealth takes 5 – 10 minutes – after that PicnicHealth’s team collects your child’s longitudinal medical records on your behalf, securely upload them to your PicnicHealth account, and then de-identifies these records for ASMD researchers.
Visit picnichealth.com/asmd-wylder-nation or email email@example.com with any questions.
NPC Sibling Study
NNDPF, APMRF, Firefly Fund, and NP Canada have chosen to partner with the International Niemann-Pick Disease Registry (INPDR) moving forward and to continue the NPC sibling study through the INPDR. The INPDR is the Niemann-Pick specific patient registry which is led and governed by the Niemann-Pick community. We are recommending that all NPC families who have shared their loved one’s data with AllStripes have their data transferred to INPDR. This is completely voluntary and each family’s decision is respected. We have worked with AllStripes and the INPDR to ensure that this is a user-friendly and fairly simple process for families.
For information on how to easily share you loved one’s records with the INPDR, or if you have any questions, please contact us at firstname.lastname@example.org.
Clinical Trial Updates
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.
Update from IntraBio: IntraBio Ltd. is pleased to confirm that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301). Read the complete announcement.
Update from IntraBio: IntraBio Ltd. is pleased to share that IB1001-301 has enrolled over 70% of the target number of patients in the second month of recruitment. Recruitment is expected to be completed by December 2022. Read the complete announcement.
Community News Updates
Update from Mandos Health: Mandos Health has shared the following November 2022 update with the NNPDF. Read the complete update.
Update from KemPharm: KemPharm has shared the following news release with the NNPDF, reporting their third quarter 2022 results. Read news release.
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.
If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters!
THANK YOU to Julie Patenaude, Kit Neal Hensler, Charlotte Gibier, Jeff Padden, Donna Norberg, Joyce Gurtatowski, JoAnn Williamson, and Anne OConnor-Smith who recently hosted Facebook Fundraisers for the NNPDF! We truly appreciate your support!
Want to host your own Facebook Fundraiser? It’s easy! Visit facebook.com/fund/NNPDF to get started!
Surveys, Studies, & Market Research
Be sure to check out our Surveys & Market Research webpage for current survey and study opportunities in the Niemann-Pick disease space. Participating in surveys and studies is important to the advancement of health options for our Niemann-Pick community members. Current surveys and studies are listed below. Contact Laurie Turner at email@example.com for any questions.
Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.
Click here to update or enroll today!
For assistance contact Laurie at firstname.lastname@example.org or call 920-542-4038
Emergency Hardship Program
The NNPDF Emergency Hardship Program continues to offer assistance 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. Click here for complete details and eligibility information.
Please contact Laurie Turner, Family Services Manager at 920-542-4038 or email@example.com if you have any questions about this program.