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Hello NTSAD,
Last Thursday, we came together for a truly unforgettable night filled with conversation, connection, and celebration. Our heartfelt thanks are extended to each of you who contributed to the success of Imagine & Believe.
It was wonderful to reconnect with old friends and make new ones, honor the Buryk family for their remarkable contributions to advancing research, and raise much-needed funds to support NTSAD's mission. Together, we are making significant strides in the global fight against Tay-Sachs, Canavan, GM1, and Sandhoff disease.
Funds raised at Imagine & Believe help us continue to drive research forward, provide support to those who need it most, and honor the legacy of those we’ve lost to these devastating conditions. We are deeply grateful for the support of our community—from the individuals and families we serve to our donors, volunteers, Board of Directors, scientific advisors, and corporate partners. Thank you all for your unwavering dedication and vital support!
Warm regards,
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Kathleen Flynn, CEO, NTSAD
P.S. And don’t forget—our online auction will remain open until November 14th! Whether or not you attended Imagine & Believe, you can still support NTSAD by bidding on some fantastic auction items. Happy bidding!
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We Surpassed Our Goal for the Evening! | |
We are deeply grateful to the NTSAD community, volunteers, and all rare allies for supporting Imagine & Believe. Together, we raised nearly $145,000. As the online auction continues, we ask for your support in helping us break the $150,000 mark by making a donation or bidding on our online auction items by 5:00pm ET on Thursday, November 14. | |
Be sure to like and follow NTSAD on social media for the most current rare community news and information. | |
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2025 NTSAD Research Initiative:
Request for Proposals is Open
Due to NTSAD's commitments to support ongoing GM1 and GM2 research projects at NIH and UMass, respectively, this year’s request for proposals is focused on research on Canavan Disease only.
We are interested in all aspects of therapeutic discovery. Basic research and translational studies are strongly encouraged to generate strong preliminary data to enable major funding by other third parties in the future.
The 2025 Request for Proposals are available now under the grant opportunities tab on the NTSAD website. Letters of intent are due by 5:00pm ET on December 2, 2024.
Questions related to the Research Initiative Program and the application process may be directed to Valerie Greger, PhD.
Visit here to see past recipients and their funded research over the years.
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NTSAD, the leading patient advocacy organization dedicated to Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff diseases, has supported rare families and advanced research for over 67 years. Since it was established in 2002, NTSAD’s Research Initiative Program has awarded more than 75 grants totaling more than $5.1 million. The data generated from research funded by NTSAD has been leveraged to attract more than $30 million in follow on grants from larger funders, including the National Institutes of Health (NIH). | |
FDA's Rare Disease Innovation Hub Meeting - October 16, 2024
The Reagan-Udall Foundation hosted a hybrid meeting where hundreds of advocates participated in a robust discussion on advancing rare disease therapies.
Kathy Flynn, NTSAD's CEO, attended the meeting at the U.S. Food and Drug Administration headquarters and and met with several other rare disease advocates.
The goal of the meeting was to bring together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss how the recently announced Rare Disease Innovation Hub should prioritize its work and best engage with members of the rare disease community.
EveryLife Foundation takeaways here.
Watch the recording here.
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Attention:
GM2 and Canavan Families
Help advance prenatal screening by participating in this study conducted by Billion to One.
This study is confidential.
If you have one of the variants shown in the chart on the right, click here to find out how to participate.
Thank you for helping!
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Advocacy Alert: Take Action to Save the Pediatric Priority Review Voucher Program
From EveryLife Foundation:
Further Congressional action is urgent. With the current Rare Pediatric PRV Program now set to expire on December 20, 2024, there is a palpable risk of losing a pivotal incentive mechanism that has encouraged the development of treatments for diseases that predominantly affect children.
We urge advocates to ask their Senators to support the Creating Hope Reauthorization Act to prevent a program that has already profoundly impacted the rare disease community from lapsing.
Click the image to the right, enter your information, and you will automatically see a generated message for legislators in your state. Easy as 1, 2, 3.
Thank you!
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Save the Date for the NTSAD
47th Annual Family Conference!
Join us in Dallas, Texas April 24-27, 2025 for the 47th Annual Family Conference, a weekend of support, connection, research, and more.
Stay tuned for more information coming soon, including an all-new Helping Hand program, and hotel reservation and conference registration links.
Contact Diana Jussila, Director of Family Services, here if you or your company would like to sponsor the 2025 Annual Family Conference.
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This Holiday Season...
The holiday season means connecting with friends, family and those we care most about. But we know the expectations of the holiday obligations can often add more stress to the many stresses already weighing on rare disease families. You may need to ask for help, say no, go slow, do less, or do things differently than those around you. So here are some articles with tips for navigating the “normal” holiday expectations as we continue to deal with the unique demands of rare disease life.
Click on the box to the right to learn more about tips for navigating the holiday season while coping with rare disease from our friends at Global Genes.
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Mission Moment: The Dragon Brothers, Luke and Isaiah
"Luke Michael and Isaiah James Thomas Ackerman were each other's wrestling partners and as thick as thieves. Both of them loved being read to and listening to music, although each had very different styles they liked. Luke was a Johnny Cash boy and Isaiah loved Bruce Springsteen. They enjoyed being around people and liked to cause some occasional mischief on an unsuspecting victim. Their hearts were filled with love for anyone and everyone they came in contact with. They just loved to be present in whatever was going on.
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My hope for their memory is that it would not be a sad story when they’re spoken of. I want their story to be one of courage and hope for those looking for answers and a cure and that they were a pivotal part in making that happen." - Debbie, Mom to Luke and Isaiah, Juvenile GM1 | | |
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Staff
Kathy Flynn, CEO
Becky Benson, Family Support & Engagement Manager
Mackenzie Briggs, Administrative Assistant
Valerie Greger, PhD, Research Director
Diana Jussila, Director of Family Services
Cynthia Perreault-Micale, PhD, Research Manager
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