News & family stories

Research breakthroughs, community voices, and updates from the movement.

Featured • Research

A gene therapy we funded just entered its first human trial

After a decade of lab work, a treatment targeting one of the most common X-linked genes has reached the clinic for the first time.June 12, 2026 • 4 min read

“The helpline answered on the hardest night”

One parent shares what it meant to reach a real person at 2am, three days after their son's diagnosis.May 28, 2026 • 6 min read

What the new newborn screening bill means

A plain-language guide to the legislation that could bring earlier diagnosis to thousands of families.May 9, 2026 • 3 min read

Meet the families walking 100km for research

This spring, forty families are walking the coast path together to fund the next round of Discovery Grants.April 22, 2026 • 5 min read

Five questions with this year's fellowship winners

We sat down with the three early-career scientists joining the field through our fellowship program.April 3, 2026 • 7 min read

How one school built a truly inclusive classroom

A head teacher explains the small, practical changes that made the biggest difference.March 18, 2026 • 5 min read

Our 2025 impact report is here

Where your support went last year — research funded, families reached, and what comes next.March 1, 2026 • 2 min read