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

Mitochondrial DNA depletion syndrome: developing a new treatment

Date: 3 September 2018 - 2 September 2021

Craniosynostosis: improving treatment for children

Date: 16 October 2017 - 15 October 2019

Previous research

Pelizaeus-Merzbacher disease – finding new treatments

Date: 2 January 2018 - 1 January 2019

Personalising surgery for babies with a serious heart disease

Date: 1 September 2015 - 28 February 2018

Creating new ears and noses for children who need them

Date: 15 September 2014 - 31 December 2016

Smith-Lemli-Opitz syndrome: working towards a new treatment

Date: 15 November 2012 - 1 November 2016
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