Advocacy
Our 2025 impact report is here
Where your support went last year — research funded, families reached, and what comes next.
In 2025, supporters like you funded eleven new research projects, answered more than 9,000 helpline calls, and welcomed 600 new families to the registry. Our full impact report breaks down exactly where every pound went.
The headline: for the first time, a therapy we seed-funded reached human trials. None of it happens without the community behind it — monthly donors, fundraisers, volunteers, and the families who share their stories so others don't feel alone.
Read the highlights, then tell us what you'd like us to focus on in the year ahead. This is your foundation as much as anyone's.