ATRX Research Alliance

Big News!

A treatment initiative for ATR-X syndrome is already underway- and we are working to fully fund the next critical phase.

The ATRX Research Alliance is raising $350,000 to fund Phase 1 of a gene therapy initiative focused on advancing a potential treatment for ATR-X syndrome, a rare genetic neurodevelopmental disorder.

This effort is the result of years of family advocacy, scientific collaboration, patient registry development, and bio-sample collection.

Today, the science is moving forward through a collaborative effort involving Dr. Allison Bradbury at Nationwide Children’s Hospital, Dr. David Picketts at the Ottawa Hospital Research Institute and the ATRX Research Alliance.

The research program is active now, including:
• Ongoing laboratory work
• Development and testing of gene therapy constructs
• Maintenance of a growing mouse colony critical to the research process

Phase 1 represents the proof-of-concept stage needed to determine whether this therapy can advance toward clinical trials.

Rare disease communities often wait decades for momentum like this. We have an opportunity to help move this science forward now.

We invite you to support this work by donating, sharing, or helping us connect with others who believe in accelerating rare disease research.

https://give.rarevillage.org/give/448928/ #!/donation/checkout

*Note: We use GoFundMe Pro, provided to us through our partners at the Rare Village Foundation. All funds donated through this platform go directly to the ATRX Research Alliance to support research initiatives.

When we gather for this conference, something powerful will happen.

Families will connect.
Researchers will listen.
Momentum builds.

But I know that at times, travel costs can make attending feel out of reach.

That’s why we created Community Connection Grants, to help ensure that cost is not the reason someone misses being part of this community.

These grants will cover 3 nights at the Renaissance Columbus Downtown Hotel during the conference.

🗓 Applications close March 15
📩 Award letters sent March 20 (giving a full month to plan travel)

If attending has been on your heart, please apply.

We want you there. 💙

After completing your conference registration, apply for the grant here:
https://atrxresearch.org/2026-scientific-family-conference/