Hello NTSAD,

Thank you to the NTSAD community, researchers, and our generous sponsors for coming together to make the 47th Annual Family Conference in Dallas such a meaningful and inspiring event.

It was truly heartening to see individuals and families connect, share their journeys, and find strength in one another, while also gaining valuable insights from leading scientists and experts. From research updates to the advocacy and caregiver support sessions, the conference offered powerful opportunities to learn, grow, and feel supported.

We are deeply grateful for your presence and passion, and we look forward to continuing these important connections and advocacy efforts throughout the year.

Warm regards,

Kathleen Flynn, CEO, NTSAD

NTSAD Annual Family Conference - What A Weekend!

For the past 47 years, NTSAD's Annual Family Conference has provided individuals and families living with Tay-Sachs, Canavan, GM1 gangliosidosis, or Sandhoff disease the opportunity to come together for precious moments to learn, lean on each other and share laughter and a few tears. Our time in Dallas was no exception. Our small but might community showed up strong! Highlights included:

246 attendees
Eight different countries represented including the U.S.
11 first-time families/individuals attended
An insightful New Attendee Orientation Session eased new families in
36 attendees of Camp Snuggle and Camp Active children living with Tay-Sachs, Canavan, GM1, or Sandhoff
Camp Sunshine and Youth Group for 25 siblings
A robust Research Day featuring an industry panel discussion and breakout sessions for each disease to learn more about studies, research, and trials
Advocacy Session that encouraged individuals and families to "Raise Their Voice"
A Texas BBQ and an incredible country music concert
Health and Symptom Management Session for families caring for younger children
Sessions for adults living with Late Onset Tay-Sachs or Sandhoff diseases
Group-specific support sessions
And time to simply connect and support one another in the quiet in-between spaces

To learn more about the NTSAD Annual Family Conference, visit our website here.

Annual Family Conference Feedback Survey

What an amazing conference it was! Thank you so very much for being a part of the 47th Annual Family Conference!

If you were in attendance with us, we ask that you please take a moment to fill out our Feedback Survey. We have a diverse group of families and individuals with a wide range of needs and preferences. We’re always working to serve you better, and our goal is to do the best we can for everyone in attendance. Your input helps make the conference the best it can be!

Take the Survey

Thank You to our 2025 Annual Family Conference Sponsors

The Annual Family Conference experience is made possible by the generosity of friends, supporters, and industry partners. We gratefully acknowledge the sponsors of our Annual Family Conference.

View the list of sponsors here.

Advocacy and Awareness

Late Onset Tay-Sachs highlighted in BridgeBio's On Rare podcast episode

The latest episode of On Rare was debuted at the NTSAD Annual Family Conference. In this episode, hosts David Rintell and Mandy Rohrig speak with Katie and Allie, who share their experience with late-onset Tay-Sachs, and Diana Jussila, Director of Family Services at NTSAD.

The episode explores how Tay-Sachs, typically diagnosed in childhood, can present in adulthood, the emotional toll of navigating a progressive rare disease, and the resilience of a close-knit sibling duo who’ve turned advocacy into action.

Listen on Apple Podcast, Spotify, YouTube, Podbean, or wherever you get your podcasts.

NTSAD Patient-led Listening Session Approved by the FDA!

We are thrilled to announce that the FDA approved NTSAD's request to host a listening session to focus on the juvenile onsets of GM1, Tay-Sachs, and Sandhoff diseases.

Our goal is to highlight and focus on the patient experience from diagnosis to the progression of symptoms. We hope the FDA will take what they learn into consideration when reviewing trial protocols and the harmful and irreversible nature of placebo-controlled trials in this group of ultra-rare and terminal diseases.

Call to Action to the FDA

During the NTSAD Family Conference, we heard about many experiences of people who have come off of Tanganil as it has become less available.

There is a drug called Aqneursa that has been approved for Niemann Pick Type C that is the more active ingredient in Tanganil. We hope the FDA will approve its use for GM2.

We share this GM2 Advocacy Call to Action with all members of our community for the opportunity to voice support to demand the FDA hold an Advisory Committee of independent experts to consider the benefits/risks of AQNEURSA for GM2.

We are taking action and need your support and input!

Take the Survey

Family Support

Join the NEW Canavan Family Support Group on Facebook!

Welcome, Canavan families!

This compassionate, supportive space is where families navigating a Canavan diagnosis or loss can connect, share, and find strength together. If you are a Canavan family member, consider joining this new group.

All families coping with a Tay-Sachs, Canavan, GM1, or Sandhoff diagnosis are welcome to join the NTSAD Family Support Group on Facebook as well.

Join the Canavan Group

Mother's Day Is

Sunday, May 11, 2025

"Whatever this (Mother's) Day does for you, whether you observe it or not, hopefully you experience many moments of peace and hope. And that the love you have for your child or children fills you with the strength and fuel of a thousand suns, to keep going, to keep fighting, no matter the circumstances."

~ Jo Kaur, Riaan Research Initiative

Did You Know?

International Bereaved Mother's Day is May 4, 2025

We recognize the moms who are experiencing this Mother's Day for the first time without their child in their arms - and those who are familiar with the ache in their heart. Carlie Marie Dudley from Australia started International Bereaved Mother's Day in 2010, in honor of her stillborn son, Christian. Since its inception, the day has helped bereaved mothers around the world connect, heal, and find hope for their future.

Ways to support a bereaved mother and all rare moms:

Say the child's name. Remind them that you remember them.
Share a special memory of their child.
Ask them to share their favorite memories.
Listen and don't minimize their feelings of grief.

International GM1 Gangliosidosis Awareness Day

Friday, May 23, 2025

International GM1 Gangliosidosis Awareness Day is a worldwide movement. The goal of this day is to bring recognition for GM1 gangliosidosis to broaden awareness of this devastating and fatal disease.

Share your GM1 stories with us by sending them to Becky at becky@ntsad.org, and we'll share them on that day!

Learn More Here

Mission Moment: Meet Debriell

Debriell F. is 16 years old and lives with GM1 Gangliosidosis, Type II. She and her mom recently attended the Annual Family Conference in Dallas, Texas. We were thrilled to have her and her mom join us!

Debbie shared that Debriell "loves hair massages while gazing at the stars. She also loves binge watching TV shows, like Full House and Moesha. Debriell loves to dance and spend time with loved ones."

Debbie, mom to Debriell, GM1

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