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
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.
Transforming lives
Support researchers as they develop an innovative new gene therapy that could transform the lives of children living with progressive familial intrahepatic cholestasis type 3 – a rare disease that, over time, causes the liver to fail.
Transforming babies’ lives
Help to fund research that could revolutionise screening of newborn babies for cytomegalovirus (CMV) – allowing early detection and treatment that can protect babies from the devastating consequences of this virus.
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.