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Hello NTSAD,
On behalf of the entire NTSAD community, I extend my heartfelt gratitude to everyone who attended and supported our Imagine & Believe event last month. Your generosity and commitment are vital in advancing our mission to improve the lives of those affected by Tay-Sachs, Canavan, GM1, and Sandhoff diseases. Your donations support our ongoing efforts to assist individuals and families as they navigate these ultra-rare genetic diseases, advocate for our rare community, and accelerate research that moves us closer to a future where treatments and cures are within reach. As we enter this season of giving, we encourage you to continue your support—whether by making a donation or spreading awareness. Every contribution, no matter the size, has a meaningful impact on the individuals and families we serve. Together, we can make the unimaginable possible. Thank you for being an essential part of our extraordinary community.
Warm regards,
| Kathleen Flynn, CEO, NTSAD | |
Be sure to like and follow NTSAD on social media for the most current rare community news and information. | |
All the Annual Family Conference Information at your Fingertips
Are you an affected family or individual or family member who wants to learn more about NTSAD's Annual Family Conference? Click on the photo on the right to view our new Annual Family Conference brochure, a resource for all things conference, all in one place!
Here you'll find important dates, FAQs, the newly revised Helping Hand policies, registration info, programming notes, and more.
Families will soon receive an email with more information on how to apply for a Helping Hand grant as well as the registration and hotel reservation links.
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2025 Annual Family Conference Sponsorship Opportunities
The Annual Family Conference is made possible by sponsors like you. You can support families and affected individuals by becoming a sponsor today.
Since 1978, NTSAD has presented the Annual Family Conference to provide families a safe space to support and learn from one another, to gain knowledge directly from researchers and industry members working on potential therapies, and to contribute and share their experiences to further enhance the development of clinical trials.
For questions about sponsoring the Conference, please contact the Development Office at development@ntsad.org or by phone (617) 277-4463.
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GM2 Voice of the Patient Report Published in Human Gene Therapy Journal
NTSAD is proud to share that our Voice of the Patient report from the 2024 Externally-Led Patient-Focused Drug Development meeting with the FDA and other key stakeholders was recently published in the Human Gene Therapy Journal. Spreading awareness about the urgent need for effective treatments and informing regulators about living with the ultrarare diseases we represent is an essential role we play as patient advocates. Thank you to every member of the NTSAD community whose voice was captured in this report.
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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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There's Still Time to 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."
Please contact your 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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Kick off the Season of Giving by Giving yo NTSAD on Tuesday, December 3rd!
The season of giving is upon us. Support family programs and advance research by making a donation below. We can't do this work without you. Give big this Giving Tuesday and help fund our mission for years to come.
Maximize your donation on Giving Tuesday or any time your give to NTSAD by asking your company about corporate matching gifts.
Please help us make a difference today.
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Worldwide Candle Lighting
The Compassionate Friends Worldwide Candle Lighting on the 2nd Sunday in December unites family and friends around the globe in lighting candles for one hour to honor the memories of the sons, daughters, brothers, sisters, and grandchildren who left too soon. As candles are lit on December 8th, 2024, at 7:00 pm local time, hundreds of thousands of people commemorate and honor the memory of all children gone too soon. Learn more at compassionatefriends.org.
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Mission Moment: Meet Jasmine | |
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"Jasmine is an extraordinary three-year-old who loves music, lights, massages, finger painting, and aquatic therapy. Since her diagnosis with Tay-Sachs disease on February 28, 2023, we have been on a journey of discovery, driven by our determination to find a solution and research the latest clinical trials. Although Jasmine's disease had progressed, making her ineligible for trials, NTSAD became a beacon of hope. The organization's support system has been invaluable, connecting us with experienced parents who have become experts in navigating this journey. Together, we are building a community that shares knowledge, offers emotional support, and celebrates each other's resilience.
Meeting families at various stages has been a profound inspiration and a testament to their strength, which highlights the boundless potential of the human spirit. Our community's power is immeasurable, providing comfort on the toughest days. Jasmine's spirit, resilience, and beauty captivates our hearts, and we are grateful for the love, support, and connection surrounding her. Jasmine embodies bravery, resilience, and love. Although this journey is a parent's worst nightmare, we are grateful for NTSAD's support." - Brittany, Jasmine's mom
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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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