Message from the Vice Chair
Dear NNPDF Community,
On behalf of the NNPDF we would like to first and foremost thank those that participated and supported our FDA Listening Session on April 9. The impact of statements from clinicians, patients, caregivers as well as the patient advocates provided incredible insight into the complexities of this disease and its impact on clinical research and patient symptom and disease management. During this session, we were able to outline areas that we, as a community, need the help of the FDA.
If you have not already seen the FDA Listening Session Summary Report, I encourage you to visit our website and read through it. It is also available in this newsletter.
As the national patient advocacy and family support organization dedicated to supporting and empowering patients and families affected by Niemann-Pick disease, the NNPDF is continuing to strategize with community members, advocates and researchers. We are finding incredible value in hearing your voices and bringing forth the importance of a united community. It is our job and our duty to remain unbiased in our work toward educating, collaborating and supporting research, so that our community can find promise in multiple therapies.
This is a very important time for our community. Patients and caregivers have all been faced with a lot over the past few months and prior. It is in these moments that we continue to work together and forward for the better of the community. We ask that we stand together, working toward a common purpose. We will PERSEVERE and continue to drive for the positive progress we can make for the lives of those affected with Niemann-Pick disease. As our community works together we will remain transparent in our strategy and continue to convey the needs of this incredible community
Becky McGuire, Cousin to Kelly – NPC
FDA Listening Session on Niemann-Pick disease Summary Report
NNPDF hosted an FDA listening session on Niemann- Pick Disease on Friday, April 9th. The listening session was a platform for the NNPDF to present to the FDA/CDER and to share the concerns and priorities from the entire Niemann-Pick disease community. To review the NNPDF FDA Listening Session Summary Report, please click here.
The Listening Session Summary Report was submitted to the FDA along with several patient voice statements and the arimoclomol support letter which was signed by 500+ Niemann-Pick community members! Thank you for supporting your Niemann-Pick community!
NNPDF-INPDA Family Support & Medical Virtual Conference
We continue to prepare for the NNPDF-INPDA Family Support & Medical Virtual Conference taking place July 29 – August 1st! Watch for registration details coming soon! In the meantime, remember to download the NNPDF app for connecting, updates, info, contests, and more!
NNPDF Cora Sterling Endurance Award Nominations
Do you know a great young person who shines for Niemann-Pick awareness? NNPDF is seeking nominations for the NNPDF Cora Sterling Endurance Award. This award is presented annually at the NNPDF Family Support & Medical Conference to a young person (under age 25) who shines in raising awareness of Niemann‐Pick disease in the community. Deadline for nominations is May 31, 2021.
Order Your NNPDF Gear TODAY!
It’s time to order some awesome NNPDF gear! For a limited time our NNPDF Pop-Up Online Store will be accepting orders for your NNPDF Tote Bags, Towels, Gaiters, T-Shirts, Jackets, Hats and more! Get some great additions for your summer outings! (US Only) Open through May 16th.
ASMD Accelerate Study
Wylder Nation Foundation has recently partnered with PicnicHealth to launch the ASMD Accelerate study as a way for families to easily contribute to advancing research for ASMD.
The study is currently enrolling both patients with Infantile Neurovisceral ASMD (NPA) and Chronic Neurovisceral ASMD (NPA/B).
There are two “caregiver” scenarios for enrollment:
1) Those who are currently on the medical journey and caring for a child with NPA or NPA/B
For these families, participation will not only help researchers better understand the disease but will also provide them with digital access to all of their medical records in one central location and give them the ability to share records with whomever they feel necessary such as other doctors or specialists.
2) Those whose child has unfortunately passed away from NPA or NPA/B
For these family’s participation is equally important to helping researchers better understand the disease and can serve as a way to carry on their child’s legacy and hopefully improve outcomes for future children diagnosed with the disease.
Enrolling in the study is easy and can be done via the study landing page. Once enrolled all you will need to do is provide a list of the doctors and/or care facilities your child has visited, and Picnic Health will do all the work of obtaining the medical records, structuring them, and uploading them to the family’s personal timeline. Click here for additional information and enrollment.
To review the recent ASMD Accelerate Study Webinar click here.
Thank you to all that have recently updated their membership. We are continuing to request that ALL current and interested community members confirm or update your membership information to ensure we have accurate contact information for you and your family for continued important notifications from the NNPDF.
For enrollment assistance contact Laurie at firstname.lastname@example.org or call 603-413-8707
Newborn Screening Update
The Newborn Screening Working Group will hold its first virtual meeting in 2021 on Friday May 14th at 4pm ET. If you are interested in learning more about the efforts of the Newborn Screening working group and the progress that is being made towards realizing the goal of having NPC added to the RUSP (Recommended Uniform Screening Panel), or if you would like to join this important advocacy and research project, please contact Pam Andrews at Firefly Fund (email@example.com) to be added to the distribution list for future communications.
Comprehensive Care Centers
NNPDF has created a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. Click here to learn more!
If you are connected with a medical team that would like to be added to the NNPDF Comprehensive Care Centers listing, please contact Laurie Turner at firstname.lastname@example.org.
Coffee & Catching Up
A Book for Children With Rare Disease
NNPDF is excited to share to that Extraordinary! A Book for Children with Rare Diseases, written by Evren Ayik and his Mom, Kara, is now available for purchase online. This book, written as an expression of support for children who live with the challenge of growing up with a rare disease, captures the lessons Evren learned beginning at age three when he was diagnosed with ASMD. Click here to learn more. Congratulations to Evren and Kara! Well done!
Emergency Hardship Program
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 $1000 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.
Patient AirLift Services
Patient AirLift Services is a nonprofit that arranges free flights for medical patients requiring medical diagnosis, treatment or follow-up who cannot afford or are unable to fly commercially.
PALS also arranges volunteer flights for family members of patients as compassionate missions, to ensure patients have support when they are away from home for long periods. PALS is proud to assist military personnel and their families with free flights to aid in the recovery and rehabilitation processes for our wounded veterans. Click here for more information.
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.
Updates from Cyclo Therapeutics: Cyclo Therapeutics announces their design of pivotal Phase 3 study evaluating Trappsol® Cyclo™ in Niemann-Pick Type C1 patient enrollment is on track to commence in Q2 2021. For complete announcement click here.
Cyclo Therapeutics announces their Phase III pivotal trial using Trappsol® Cyclo™ will be enrolling soon. For complete announcement click here.
Update from Sanofi Genzyme: Sanofi Genzyme has shared an update on compassionate use program for olipudase alfa. For complete announcement click here.
Illustrate Your Imagination
In recognition of Global Niemann-Pick disease Awareness Month, the Orphazyme team invites you to join us on a quest to “Illustrate Your Imagination.”
Our team often hears brave stories of your experiences living with Niemann-Pick disease and times when you took on challenges with deep personal courage, perseverance, care, and integrity. We’d like to help your super stories take flight.
Your mission, should you choose to accept it: Visit IllustrateYourImagination.com and download a kit to create your own comic. The kit is filled with page templates, instructions, and some flair to help bring your story to life. Submit your story to our online comic book gallery and check out submissions from other superheroes around the world.
We can’t wait to see the creative stories you come up with!
Head of Global Patient