Dear Family and friends,

As 2024 comes to a close, we are filled with gratitude for the support that has carried us through this year. What began just a year ago as a deeply personal mission for our 2-year-old Lukie has now grown into a shared vision for a brighter future for families affected by ZTTK.

Since joining the ZTTK SON-Shine Foundation as board members and leaders, we’ve mobilized the ZTTK community to drive research. Thanks to your support—through donations, sharing with your networks, and other acts of kindness—we have raised nearly $1.4 million to-date, including an $800,000 grant from the Chan Zuckerberg Initiative!

Your support has enabled us to:

1.  Accelerate ZTTK research: We’ve made significant progress, including developing the first ZTTK stem cells, launching a patient database, initiating a metabolomics study, and funding a drug repurposing project, with more efforts underway.
2. Identify more patients: Sharing our story has helped identify more ZTTK patients globally and expand our community.
3. Develop a patient-centered research agenda: We are planning our inaugural global ZTTK Conference in Boston next summer to convene researchers, clinicians, and families.

None of this progress is possible without your support. As we look ahead, we remain dedicated to advancing research, advocating for patients, and supporting Lukie and his ZTTK friends. This season of giving, your year-end contribution can help sustain our momentum and bring us closer to life-changing breakthroughs.

Happy Holidays and sending you lots of sweet Lukie’s smiles!

With heartfelt thanks,
Lukie, Ada, Nathan

Dear Lukie’s Supporter,

Happy Fall! Lukie continues to be busy scooting everywhere and going on new adventures!

Since we started our rare disease journey last year, we are now connected with 250+ ZTTK families around the world, and we are leading the ZTTK SON-Shine Foundation to accelerate ZTTK Research.

Your support is making a real impact for Lukie and his ZTTK friends, and we’re thrilled to share the latest updates:

$800,000 Grant from the Chan Zuckerberg Initiative

We’re honored to receive a grant from the Rare as One project to grow our research partnerships and develop patient-focused treatments. This is a huge milestone for our mission!

5K for ZTTK & September Awareness Month

With 370+ participants across the U.S., Portugal, Australia, and beyond, the 5K for ZTTK raised over $45,000 for ZTTK research. Events like this will also help us raise awareness globally, and help us identify and connect with other ZTTK families

Research Updates

New partnerships with Citizen Health, Rare-X, and a Boston biotech will expand patient data and accelerate treatments. We’ll keep you posted on our progress!

Upcoming 2025 ZTTK Family & Science Conference

Plans are underway for our 2025 conference to convene families, clinicians, and researchers to support ZTTK Families and advance research.

If you’d like to help with fundraising, event planning, or sponsorship, please email Ada Lio at alio@zttk.org.

Once again, thank you for all your support. Together, we’re paving the way for breakthroughs in ZTTK research and care.

With Gratitude and Hope,
Lukie, Ada, Nathan

Hi Lukie’s supporter!

We hope you’re enjoying the start of summer! As the days get warmer, we’re thrilled to share some updates from the past few months and invite you to join us in our ongoing journey.

Lukie’s Adventures
Lukie has been quite the little explorer lately! From visiting Dallas and Acadia to tackling his daily therapies and hospital visits, he’s been a very busy toddler. This summer, Lukie is ready for more adventures and to hit the pool for some swim lessons!

Fundraising Fun
We’ve got a lineup of exciting events this summer and beyond, and we’d love for you to join us:

1. June 22, Saturday (5-7pm): Pre-10K Cheers for ZTTK Research
If you’re local, come by the Aeronaut Beer Garden in Allston. We’ll have a table to raise awareness for ZTTK and limited editions of Lukie T-shirts and stickers for ‘Lukie Ambassadors’! More event details here

2. June 23, Sunday (8-9am): Boston BAA 10K
Cheer for Ada as she runs to rare awareness and funds for ZTTK Research. Wear your Lukie T-shirt and join us along the course! Course map here

3. September – ZTTK Awareness Month:
We’re planning a mix of in-person and virtual events. Stay tuned!

We’re incredibly thankful to those of you who have organized fundraising activities on Lukie’s behalf. If you’re interested in hosting a fundraiser or event for Lukie, please reach out to Ada at ​alio@zttksonshinefoundation.org

Science Updates
In the last few months, we have kicked off projects that are vital steps toward our ultimate goal of developing effective treatments for ZTTK:

ZTTK Stem Cell (iPSC), Mouse Model, ELISA 
We kicked off three projects with dedicated scientists to accelerate drug development. These projects will allow us to better understand disease mechanisms, screen candidate drugs, and test new treatments

First ZTTK Patient Registry
We continue to work on aggregating patient data through Rare- X to provide deeper insights into disease progression and symptoms, paving the way for more targeted research and treatments.

We will continue to explore additional approaches and partnerships to balance the urgency of accelerating drug development with achieving the highest return on investment.

Your support is the backbone of our mission. Every dollar, every shared story, and every event hosted makes a significant impact. We are incredibly grateful for your involvement and look forward to many more adventures together.

Thank You!
Warmly,
Ada, Nathan, and Lukie

Hi Lukie’s supporter!

It’s been a very busy Rare Disease week and start to 2024! ZTTK is getting a lot of great press to drive awareness and fundraising – Lucas’ story made both the front page of the Boston Sunday Globe and CBS News Boston! We greatly appreciate all of your continued advice and support!!

Some updates!
Fundraising
1) >$150K fundraising since launch in December ’23. We are now fundraising out of a new GoFundMe that links directly to the SON-Shine Foundation (We joined the Board officially in Jan!)
2)  Held two local Somerville fundraising events during Rare Disease Week. Building local community awareness including upcoming events.

Science
1) Launched first ever ZTTK patient registry to aggregate patient data to better understand disease progression and symptoms
2) Held Q1 ’24 ZTTK Scientific Advisory Board (SAB) Meeting
3) Ahn Lab ZTTK mouse model paper in-press review
4) Accepted by Jackson Laboratory to develop mouse model with the most prevalent SON variant (c.5753_5756del, p.Val1918Glufs*87) funded under the existing grant

What’s next? 
1) Finalizing ZTTK iPSC project plan and targeting project kickoff in March/April.
2) Evaluating approach and partners for the following projects:
– Genotype-phenotype data aggregation and analysis
– Biomarker / assay project(s)
– Small molecule repurposing project

Thank you for your continued support and for sharing our efforts with your networks.

Sincerely,
Ada, Nathan, Lukie