Targeting rare diseases in children | Action Medical Research

Targeting rare diseases in children

Thousands of families across the UK are coping with the challenge of caring for a child with a rare and devastating disease for which there is no cure. With your help we can develop therapies for diseases in children including the genetic disorder Smith-Lemli-Opitz syndrome, rare forms of brain cancer and blindness, as well as startle disease.

Current research

Craniosynostosis: improving treatment for children

Date: 2 January 2018 - 1 January 2020

Personalising surgery for babies with a serious heart disease

Date: 1 September 2015 - 28 February 2018
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