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 Danny who has the rare disease, Hunter syndrome. Danny’s family are painfully aware that time with their youngest son will be cruelly cut short by this disease.
“We will lose our beautiful boy to Hunter syndrome,” says mum Sally. “But any new hope is worth fighting for. So that families in the future don’t have to feel that the bottom is dropping out of their world.”
Help fund vital research – together we can bring hope to families fighting rare and incurable diseases.
Without a lung transplant, less than one in five children born with lung surfactant deficiency, a rare lung condition, will live to celebrate their fifth birthday. With your help this Christmas, we can support research that could change this and help save babies' lives.
Support research that offers hope of curing X-linked lymphoproliferative disease (XLP) – a devastating condition that primarily affects boys and puts them at serious risk of developing life-threatening complications.
Help fund a cutting-edge piece of research that could improve the outlook for children fighting a rare brain disease, called NMDAR-antibody encephalitis. A disease that can have a devastating impact on young lives.
We need your help to fund research to better understand how COVID-19 affects babies, children and young people and help tackle COVID-19 in children.