Dear Family, Friends, and Partners of CTNNB1 Connect & Cure,
As we step into 2026, we’re carrying forward real momentum built by you.
This last quarter reflects that progress across research, community, and the systems that support this work, made possible by you.
In Q1, you helped:
- Advance multiple therapeutic strategies in parallel
- Move drug repurposing into next phase
- Fund continued small-molecule drug development
- Expand mRNA research
- Center patient and caregiver voices in research priorities
Research Updates: Progress in Motion
Small-Molecule Therapy Development
This past quarter, we awarded an additional $50,000 to Dr. Michele Jacob’s lab at Tufts University, extending critical work on a potential small-molecule therapy.
The collaboration with Tufts, NCATS (NIH), and the Broad Institute continues to make encouraging progress, with improved chemistry breakthroughs supporting the goal of identifying a viable drug candidate within the next 12–18 months.
mRNA Booster Research Expands
Research led by Dr. Jeffrey Coller at Johns Hopkins University is advancing exploration of mRNA boosters designed to support CTNNB1 function.
The team is actively evaluating multiple candidates to determine which show the strongest therapeutic potential. This approach represents an important expansion of our research portfolio and a promising avenue toward targeted treatment strategies.
Drug Repurposing: Moving to the Next Phase
Following a comprehensive review of 28 candidate drugs, our Medical Scientific Advisory Board has completed initial prioritization.
This quarter marks the transition into deeper analysis with clinical pharmacology expertise to evaluate:
- Biological relevance
- Safety and dosing
Drug repurposing remains one of the most efficient potential paths to treatment due to existing human safety data.
Registration is officially OPEN for the 2026 CTNNB1 Connect & Cure Conference
Boston | October 8–10, 2026
We’re excited to bring our community together for research updates, collaboration, and connection between families, researchers, clinicians, and advocates. We hope to see you there!
Disease Concept Model: Your Voices in Action
Caregivers in the CTNNB1 community are helping shape the future of research.
Through work led by Sophia Marek (Rutgers University), insights on daily challenges and priorities have been captured and analyzed.
Findings will be shared at the October 2026 CTNNB1 Connect & Cure Conference, helping ensure future therapies reflect what matters most to families.
Organizational Growth
Welcoming New Board Leadership
We welcomed Anita Chang (CFO) and Ashley Hamic (COO & Secretary), bringing financial and operational expertise to support our continued growth.
Community Spotlight: Ava
We celebrate Ava’s recent milestone of graduating from high school.
She continues to build independence through a transition program and is planning to join an in-person program next year. Ava’s journey reflects the power of persistence, support, and growth over time.
Her story reminds us that progress isn’t always measured in big leaps but in meaningful steps forward.
Fundraising Impact: Community Power
The Dreckman Family led an extraordinary golf fundraiser:
- 120 golfers and 30 supporters
- Nearly $35,000 raised
We are so thankful for their support and commitment.
Their effort is a powerful example of how community-driven initiatives fuel real research progress.
Awareness & Advocacy Highlights
- CTNNB1 was represented in the Genetic Causes of Cerebral Palsy webinar series
- Community members were recognized in the Cerebral Palsy Research Network Photo Contest
- Continued efforts to raise visibility for genetic causes of cerebral palsy and CTNNB1
Each moment of visibility brings us closer to earlier diagnosis, better care, and stronger advocacy.
Financial Stewardship: This Quarter
Your generosity continues to be deployed with intention and discipline:
- Prioritizing high-impact research investments
- Expanding fundraising capacity to support growth
- Maintaining strong governance and accountability
We remain committed to ensuring every dollar drives measurable progress.
Looking Ahead 
One gene. Many paths. One chance to change the future.
In the months ahead, we will continue advancing our research strategy, evaluating opportunities, and aligning resources with the most promising paths forward.
Because of you, this work continues, steadily and with purpose.
Thank You
This past quarter’s progress belongs to you.
Together, we are transforming generosity into action, and action into hope for every family affected by CTNNB1 syndrome.
With gratitude and hope,
The CTNNB1 Connect & Cure Team