Fighting rare diseases
In the UK, there are thousands of children living with a rare and life-limiting disease for which there is no cure.
Children like Eva who has the rare liver disease, progressive familial intrahepatic cholestasis type three (PFIC3). With few treatments available, Eva's family knows there will come a time when her health will worsen, and a liver transplant will likely be the only treatment option.
"It feels a bit like a ticking time bomb," says mum Sophie. "The longer Eva remains well, the better in terms of her development and growth. But also, the more time there is for research and for new treatments to become available."
Help fund vital research – together we can bring hope to families fighting rare and incurable diseases.
Other appeals
Unlocking the answers to a rare brain disease
Help fund research to better understand Rasmussen’s encephalitis, the rare condition that led Lilly-May to undergo life-saving brain surgery at just four years old, and help unlock new treatments for children in the future.
Fighting premature birth
Over 1,000 babies die each year in the UK due to premature birth. Globally, it’s the biggest killer of children under 5. Join the fight for babies born too soon. Together we will find the answers.
Fighting rare disease
30% of children with a rare disease will lose their lives before their fifth birthday. With your help we can develop treatments and cures to tackle rare diseases that devastate children’s lives. Help fund vital research.