An Update on the CTNNB1 Gene Therapy Clinical Trial -

We are thrilled to share that we could be just months away from the start of the first ever CTNNB1 Clinical Trial! Thanks to the remarkable efforts of the CTNNB1 Foundation based out of Slovenia, and CTNNB1 families all over the globe, over 4 million euros have been raised over the past 4 years to get to this point.

Last summer, families traveled to Slovenia for the first ever international conference where you may recall that 80 children were evaluated for the trial in the “Dragonfly Natural History Study.”

This summer, this Dragonfly Study is relieving an enormous amount of stress from the U.S and Canadian families by taking away the burden of traveling overseas with our special kids by joining us at the U.S. CTNNB1 Conference, hosted by Connect and Cure, right here in Boston on July 10-12th. Traveling to Boston for the evaluations is much easier and more affordable for families than traveling all the way to Europe again - but we need your support!

This campaign is being created to help fund the team of medical doctors’ travel and lodging expenses to be able to join us in Boston. This will allow us to:

Ensure the medical team will be in Boston to evaluate our children
Ensure our children remain in the running for the gene therapy clinical trial
Strengthen relationships with researchers and medical teams who are championing this cause

This is a huge step in getting closer to selecting the candidates for the trial, and we are grateful to have the team from Europe joining us in Boston. But we can’t do it without you! Every dollar helps!

Thank you so much for your continued support.

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CTNNB1 Gene Therapy Dragonfly Study in Boston

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