March 2023 Newsletter
Message from the Board Chair | Family Support & Medical Conference | Conference Scholarships | Early Bird Registration Discounts | Family Advisory Working Groups | Conference Accommodations | NNPDF Cora Sterling Endurance Award | ASMD Scholarship | ASMD Survey Opportunity | NNPDF and NTSAD Community Meeting Update | The Impact of Dysarthria | NNPDF Advocacy Update | Courageous Parents Network Virtual Workshop | The Assistance Fund | Fundraising | Clinical Trial Updates | Surveys, Studies, & Market Research | NNPDF Membership | Emergency Hardship Program
Message from the Board Chair
Dear NNPDF community,
We are very pleased to announce that registration for our upcoming Family Support & Medical Conference is now open! We are excited to host our in-person conference in Orlando, Florida, from July 20-22, 2023. While COVID showed us the possibilities of virtual meetings and helped us all stay connected, nothing can compare to the amazing experience of being together in person.
For most people, and especially in rare disease communities, joining together in-person makes it possible to interact and share insights and experiences in ways that are dynamic and often very emotional and meaningful. Despite being strong, our community is small and geographically dispersed. For many, the conference has been the very first chance to connect with others who truly understand the daily challenges of living with Niemann-Pick disease. These meetings have been transformational moments for many of us. Connections from the meeting have turned into lifelong friendships and support networks.
In addition to meeting with other families, attending the conference in person provides the opportunity to fully immerse yourself in the experience and hear directly from leading experts in research and care from the scientific, medical, and advocacy communities. You will have the chance to participate in hands-on workshops, ask questions and share your opinions, interact with experts in our breakout sessions, and join for our incredibly fun gala dinner event.
To help ease the financial burden of attending this meeting, NNPDF is proud to offer scholarship opportunities that can help with some travel costs. To learn more and apply, please visit: nnpdfconf.org/scholarships.
We also understand that many people still have concerns about traveling and gathering in-person. We are taking all necessary precautions to ensure the safety and well-being of everyone who attends the meeting this year. For those who cannot attend in person, there will still be a virtual option.
We are so excited to be in the final stages of planning this special annual event and sincerely hope you will all join us to celebrate our community and learn about the advances in research and the best ways to support all people impacted by Niemann-Pick disease. For more information and to register for the conference, please visit: nnpdfconf.org. As always, we are here to answer any questions or provide support in any way we can. Please do not hesitate to reach out to us at email@example.com.
I can’t wait to see you all soon!
NNPDF Board Chair
July 20 – 22, 2023 | Wyndham Grand Bonnet Creek | Orlando, Florida
NNPDF is busy planning the details of our 31st Annual NNPDF Family Support & Medical Conference and we are excited to see you there! NNPDF has many exciting opportunities to get involved in research and to connect with expert clinicians, researchers, and other Niemann-Pick families who share your journey. Be sure to watch NNPDF communications for important updates and deadlines.
Application Deadline April 1st
Scholarship funding is available to help families attend the 2023 NNPDF Family Support and Medical Conference. NNPDF members living in the US are eligible to apply for scholarship assistance. The NNPDF Family Conference Scholarship is available for families to help ease the cost of attending the family conference. This scholarship is limited to immediate family members of an individual with Niemann-Pick Disease.
Early Bird Registration Discounts
Register by May 15th
Discounted Registration Rate:
Register by May 15th to take advantage of discounted registration fees.
Family Registration Discounted Rate:
EARLY BIRD REGISTRATION ONLY – Register 2 adults and 2 children (age 17 and younger) at the early bird registration rate of $400 (adults $150 and children $50). Any additional children in your immediate family will be free of charge.
First Time Attendee Discount:
Enjoy a 10% discount off your registration! We’re glad to have you!
NNPDF Family Advisory Working Groups
Application Deadline May 31st
NNPDF will host Family Advisory Working Groups that will take place on Thursday, July 20, 2023 from 1:00 pm – 4:00 pm EST. This is an optional event that will take place prior to the start of our Family Support & Medical Conference at the Wyndham Grand Hotel in Orlando. Family Advisory Working Groups will provide Industry partners the opportunity to connect privately with a group of 6-10 NPC or ASMD patients and/or immediate family members for a 3-hour session. Pre-registration is required. Application deadline is May 31st.
NNPDF Cora Sterling Endurance Award
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. Nomination deadline is May 15th.
Joele Ruppert & Joseph Colton ASMD Scholarship
NNPDF is accepting applications for the Joele Ruppert and Joseph Colton ASMD Scholarship created in honor of the lives of these two cherished children (both diagnosed with ASMD) and their parents. This scholarship provides one-time funding of $500.00 to individuals diagnosed with ASMD. Application deadline is May 15th.
ASMD Patient Experience with Olipudase Alfa Survey
The International Niemann-Pick Disease Registry (INPDR) is conducting this survey in collaboration with The National Niemann-Pick Disease Foundation, Inc. (NNPDF), International Niemann-Pick Disease Alliance (INPDA), and Niemann-Pick UK (NPUK). This survey forms part of a larger study that includes interviews with Niemann-Pick disease type A/B and type B (ASMD) patients, which will explore the questions in this survey in more depth.
If you or someone you know are interested in participating, learn more and take the survey here.
NNPDF and NTSAD Community Meeting Update
A community meeting which brought together the NNPDF, the National Tay Sachs & Allied Diseases Association (NTSAD) and Azafaros, a clinical-stage biotech company, was held on February 8, 2023.
During this lively Q and A session the Niemann-Pick and GM2 patient community had the chance to receive more information and ask questions to the medical team at Azafaros regarding the Phase II study – RAINBOW. This is a randomized, double-blind, placebo-controlled, multicentre, 12-week study to evaluate the tolerability, pharmokinetics and pharmodynamics of Oral AZ-3102 in patients With GM2 gangliosidosis or Niemann-Pick Type C (NPC) diseases.
The study aims to evaluate the correct doses of AZ-3102 by evaluating the clearance of the treatment from the body and the safety of two different doses in a small number of patients with these diseases.
The two organizations and Azafaros held the positive exchange as part of their mutual and long-standing goal to find treatments and solutions for GM1, GM2, and Niemann-Pick Type C (NPC) diseases.
If you would like further information on the RAINBOW study, ask your care team or go to clinicaltrials.gov. For further inquiries you can reach out to Gisela Linthorst, Head of Patient Advocacy at Azafaros at gisela.Linthorst@azafaros.com.
The Impact of Dysarthria
In this video, NNPDF Board Member Cara Gilmore provides insight into her journey with Niemann-Pick disease type C and the impact of Dysarthria on her day-to-day life. Thank you for sharing Cara. Watch the video.
NNPDF Advocacy Update: Interstate Medical Licensure Compact
The Interstate Medical Licensure Compact is an agreement among participating U.S. states to work together to significantly streamline the licensing process for physicians who want to practice out-of-state. It offers a voluntary, expedited pathway to licensure for physicians who qualify. The Compact has the potential to increase access to health care and extend the reach of physicians through telemedicine.
As a partner of the Rare & Ready Genetic Condition Coalition, NNPDF has joined with 46 non-profit rare disease patient organizations in support of the Interstate Medical Licensure Compact. Read the letter below and learn more about this issue.
Courageous Parents Network Virtual Workshop
Anyone, from infancy through adulthood can experience medical trauma. Caregivers can too. Understanding what medical trauma is and knowing the signs can help you get support for your child and family. Dr. Meghan Marsac, pediatric psychologist and pediatric medical trauma expert, and Melissa Hogan, mother of a child with a rare disease, wrote the book “Afraid of the Doctor” to help families who experience stressful and potentially traumatic medical events. Join us in conversation with them to learn more about this important topic. Register here!
The Assistance Fund
Struggling with out-of-pocket expenses due to acid sphingomyelinase deficiency (ASMD)? The Assistance Fund is an independent charitable patient assistance organization helping patients and families by providing financial assistance for the cost of treatment and other health-related services. Through its Acid Sphingomyelinase Deficiency Financial Assistance Program, eligible individuals can receive assistance for all FDA-approved treatment for ASMD. Learn more.
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 Mari Lombardo Sanetra, KayLa Miller, and Gianna Kandrach 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!
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.
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. Contact Laurie Turner at firstname.lastname@example.org 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.
For assistance contact Laurie at email@example.com 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 firstname.lastname@example.org if you have any questions about this program.