Harper's gene therapy trial: a journey of hope and urgency
Join us as we share groundbreaking news about Harper's gene therapy trial. This page provides essential details about her upcoming treatment, the significance of this opportunity, and how you can learn more and support her journey.
A groundbreaking opportunity for Harper
Harper is poised to become the third patient worldwide and the first from North America to receive this pioneering gene replacement therapy. While this is a clinical trial and not a guaranteed cure, her medical team is cautiously optimistic about the potential for significant gains in Harper's abilities. This incredible opportunity has only been made possible through the tireless efforts of parents within the global CTNNB1 community, who have collectively raised millions of dollars to reach this pivotal point.
Understanding the procedure and its urgency
Many are curious about the procedure itself and any potential risks. Harper will undergo two injections of a synthetic copy of a functioning gene, delivered directly into her brain using a viral vector. Following the procedure, her postoperative care and initial rehabilitation are expected to last between 4 to 6 months. There is a strong sense of urgency surrounding this treatment; as Harper gets older, her developmental window for benefiting from this therapy is closing. Waiting is simply not an option. We believe that with hope and a united community, miracles can truly happen.
Learn more about CTNNB1 and clinical trials
Rare diseases often struggle to attract the funding needed for research and treatment development, not because treatments are impossible, but due to the immense financial commitment required. This trial represents a beacon of hope for many. For more in-depth clinical information about CTNNB1 Syndrome and the gene therapy trial, we encourage you to visit the CTNNB1 Foundation website.
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