Never got to ride a bike
Action Medical Research is striving to develop treatments and cures to help save children’s lives. But we can’t do it alone.
With your help we will NEVER give up. Together we can fight rare diseases.
Never Give Up
Two-year old Emmy has Vici syndrome, one of the most severe multi-system conditions that can affect children.
Sadly, there is currently no cure or effective treatment for this devastating rare disease – and most affected children do not live beyond the age of five.
We were blown away by such a catastrophic diagnosis
Evan’s story
Evan was diagnosed with a rare brain cancer called medulloblastoma when he was 15 months old. Luckily, he now has the all-clear but is living with complex complications as a result of the tumour and treatment effects.
Paddy’s story
Paddy has KCNT1-related epilepsy, a very rare and severe condition that causes seizures. There is no cure and children are often left severely disabled. But most heartbreakingly of all, their lives are often cut tragically short.
Danny’s story
Danny has Hunter syndrome, a rare disease with no cure and limited treatment options. His family are painfully aware that time with their youngest son will be cruelly cut short by this rare disease.
Sophia's story
Sophia has spinal muscular atrophy type 1, a rare disease that causes progressive muscle weakness and movement problems. A new treatment has stopped the progression of this cruel disease for now, but Sophia's future remains uncertain.
Research into rare diseases in children
We are funding some amazing research projects across the UK. Research that could lead to new treatments, cures and medical breakthrough for children fighting a rare disease.