At just 8 years old, Harper has already shown the world what courage looks like.

Harper lives with CTNNB1 Syndrome, an ultra-rare genetic condition caused by changes (mutations) in the CTNNB1 gene. This gene plays a critical role in brain development, muscle tone, vision, communication, and overall neurological function. When it doesn’t work properly, children can face:

Significant developmental delays

Limited or absent speech

Low or spastic muscle tone

Balance and coordination challenges

Vision impairments

Cognitive disabilities

There is currently no cure for CTNNB1 Syndrome. Treatment has only meant supportive therapies — physical therapy, speech therapy, occupational therapy — all focused on helping children reach their fullest potential while managing lifelong challenges.

But now, something extraordinary is happening.

Harper has been selected as the THIRD child in the world to participate in a groundbreaking human clinical trial for a new gene replacement therapy designed specifically for CTNNB1 Syndrome. This innovative therapy aims to deliver a functional copy of the gene directly to the body, targeting the root cause of the condition rather than just managing symptoms.

For families affected by rare genetic disorders, this is more than a trial — it’s hope.

If successful, this therapy could:

Improve mobility and muscle strength

Support cognitive development

Enhance communication abilities

Slow or halt neurological progression

Change the standard of care for CTNNB1 Syndrome

Beyond Harper, positive trial results could open doors for the entire rare disease community. Gene replacement therapies are paving the way for treating conditions once thought untreatable. Harper’s participation contributes vital clinical data that could help accelerate research, funding, and treatment access for thousands of children living with rare genetic disorders.

Being chosen is an incredible honor — but it comes with a significant financial burden.

Over the next three years, Harper will undergo:

Specialized hospital stays

Ongoing clinical monitoring

Extensive follow-up assessments

Rehabilitation and therapy

Travel to and from the trial site

Insurance-related costs not covered by provincial health care

The total estimated cost is $250,000 to ensure Harper can complete the full three-year clinical trial and data collection process.

Every donation helps cover:

Travel expenses

Hospital and medical costs

Rehabilitation and therapy

Required follow-up assessments

Ongoing monitoring until trial completion (2029)

This is more than funding medical care.

This is investing in the future of rare disease treatment.

This is helping one brave little girl change the world.

Harper is funny, determined, and full of light. She works hard every single day to do things many of us take for granted. Now she has the chance to be part of something revolutionary.

Please consider supporting Harper’s journey. Share her story. Be part of this historic moment.

Together, we can help Harper — and children like her — move closer to a future filled with possibility.