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

Leukaemia – targeting treatment

Date: 1 December 2015 - 30 November 2018

Personalising surgery for babies with a serious heart disease

Date: 1 September 2015 - 28 February 2018

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

Date: 15 November 2012 - 1 November 2016

Previous research

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