Hello Diana,

NTSAD continues to champion the voices of those affected by Tay-Sachs, Canavan, GM1, and Sandhoff diseases. While encouraging our entire community to participate in Day of Hope, we are reminded how impactful it can be to share your personal journey to help raise awareness of the unmet needs for the rare diseases we represent.

Whether you post your story on social media; host a Move a Mile gathering at your school, office, or neighborhood park; or send a message to your local legislators – your actions shine a light on the rare diseases we represent and the ongoing need for research and support. When our voices come together, we make a difference and drive meaningful change.

Please be sure to visit our website to access the many resources available and to keep up-to-date with the latest happenings at NTSAD.

Kathleen Flynn, CEO, NTSAD

Welcome to the NTSAD Family, Mackenzie!

We're pleased to introduce our newest staff member and part-time Administrative Assistant, Mackenzie Briggs.

"Hi everyone! My name is Mackenzie Briggs, I'm the new Administrative Assistant with NTSAD. I recently moved up from Virginia to study at Simmons University in their Master of Library and Information Science Program. I'm very excited to work with NTSAD and get to know everyone!"

Please join us in welcoming Mackenzie to the NTSAD community.

Big Things are Happening in September!

✶ It is the 67th anniversary of NTSAD's official founding!

✶ September is also Tay-Sachs and Leukodystrophy month - an opportunity to raise awareness about these diseases.

✶ NTSAD launches a new series of papers about "Understanding Therapeutic Approaches" for Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff.

✶ NTSAD's Day of Hope turns 14 years old! Families have raised nearly $750,000 since our first Day of Hope!

✶ NTSAD's Fourth Regional Family Meetup will take place on September 21 and 22, in Short Hills, New Jersey, bringing local families together for connection and support.

✶ September is the month to a Move a Mile for Hope to raise funds for research for Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff!

Be sure to like and follow NTSAD on social media for the most current rare community news and information.

Understanding Therapeutic Approaches

NTSAD's research team created a series of new resources to help the community understand the biological basis of the current therapeutic approaches being explored in Tay-Sachs, Canavan, GM1, and Sandhoff diseases.

The papers cover the approaches from gene therapy to enzyme replacement therapy and can be downloaded from our website here as they are published over the next few months.

Passage Bio Announces New Home for

GM1 Gene Therapy Program

Good news for the GM1 community!

Passage Bio recently shared that the newly formed GEMMA Biotherapeutics will continue the work of the GM1 Imagine-1 gene therapy program that Passage Bio started a few years ago. Read the full press release here.

Stay tuned for more details after NTSAD connects with the new team!

Share Your Voice!

The FDA’s Center for Biologics Evaluation and Research (CBER) is excited to host a patient and care partner listening meeting – Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases on Friday, September 20, from 11:00 AM to 4:30 PM ET.

The meeting aims to gather patient and care partner feedback on gene therapy risks, useful information for decision-making, experiences with long-term follow-up studies, and to develop patient-centered protocols for ongoing gene therapy monitoring. To participate in this meeting, please register.

Attention Families in the Chicago and Washington, D.C. Areas!!!

Gameday with Chicago Red Stars on September 21:

Uplifting Athletes is excited to invite families to join us for a one-of-a-kind event with the Chicago Red Stars at SeatGeek Stadium on September 21, at 7:00 PM CT! Register to attend for free and cheer on the Chicago Red Stars as they take on the San Diego Wave! This event will include game tickets, t-shirts, food, beverages, and more! SeatGeek Stadium is ADA accessible. This Uplifting Experience is inclusive and accommodates individuals of all ages and abilities. Register today at https://bit.ly/4crKSUo!

Bowling with Northwestern Football on September 22:

Uplifting Athletes is excited to invite rare individuals and families to join them for a one-of-a-kind bowling event at Bowlero Niles on Sunday, September 22, from 2:00 to 4:00 PM CT! Register to attend for free and bowl with the Northwestern Football Team! Bowlero Niles is ADA accessible. This event will include lane and shoe rentals, an arcade card, t-shirts, food, beverages, and more. This Uplifting Experience is inclusive and accommodates individuals of all ages and abilities. Register today at https://bit.ly/3Mlc5xa!

Washington Spirit Gamedays on September 7 and September 15:

Uplifting Athletes is excited to invite rare individuals and families to join us for a one-of-a-kind gameday experience at Audi Field in Washington, DC, on September 7 and 15! Audi Field is ADA accessible. Register to attend this free event and watch the Washington Spirit take on the Houston Dash at upliftingathletes.org/events! 

What Can You Expect at NTSAD's Annual Boston Fundraising Event?

✶ A lot of heart and camaraderie as we raise money to support families and individuals living with relentless diseases like Tay-Sachs, Canavan, GM1 gangliosidosis, or Sandhoff.

✶ A time to honor the Buryk Family who have moved the needle for Late Onset Tay-Sachs research and raised awareness for all the diseases NTSAD represents.

✶ A chance to bid on a fabulous silent auction that is notorious for items such as a visit to the famous Matunuck Oyster Farm, a good selection of wine and jewelry, weekend getaways in Chicago and D.C., and a variety of unique experiences, just to name a few.

If you, your company or a company you know want to support our 67 year old mission, sponsorship opportunities range from $500 to $25,000. Deadline to be included in electronic materials is October 10.

Admission is $225 per guest. Donations and digital tribute ads are welcome.

RSVP and/or sponsor this important fundraiser here.

Seeking Auction Items & Experiences

We're seeking donations for our Imagine & Believe silent auction, including items such as:

• Jewelry
• Wine
• Theater tickets
• Sporting event tickets or memorabilia
• Hotel stays
• Dining experiences

Your generosity will significantly impact our fundraising efforts and help provide vital programs and services to affected families and individuals. All auction donors will be recognized in the digital and printed program.

If you are able to donate, please contact the NTSAD office at 617-277-4463 or development@ntsad.org with any questions, or to learn more about sponsoring or purchasing tickets to attend Imagine & Believe.

Support Research and a

Future for Families Everywhere

Join the movement that has raised nearly $750,000 to advance research! You can make a difference - whether you host your own event, support someone else's or simply donate.

Move A Mile for Hope on September 21!

Every dollar brings us closer to breakthrough treatments for Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff diseases.

NTSAD funds the research that will change lives. With your help, we can turn hope into reality.

We welcome you to join our community for Day of Hope by moving a mile with us, hosting an event or fundraiser, purchasing a t-shirt, or making a donation!

Be sure to post your photos as you Move a Mile and tag @ntsad!

Contact becky@ntsad.org today to learn more or to share how you're moving this year!

Mission Moment: Meet Tobin and Adley

Tobin and Adley are sisters both diagnosed with Canavan disease. As their mother Meagan shares below, these precious girls are full of heart and personality.

"Tobin will be 7 at the end of September and is such a happy girl. About anything can make her laugh - funny noises and anything her dad does! Tobin loves to be on the go and loves going to school.

Adley is 8 months old and also a very happy baby. Adley loves snuggles and being held and when she gets to play with her dad! They are both daddy’s girls! At this time, both girls are doing very well and staying happy and healthy! We pray it stays that way!"



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