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In the autumn statement on Wednesday 22 November, the government announced its support for the Rare Therapies Launch Pad, a new programme that will develop regulatory pathways for children with rare conditions to access individualised therapies.

The Oxford-Harrington Rare Disease Centre (OHC) in the Department of Paediatrics has a central role in the delivery of this programme, alongside Genomics England, the Association of the British Pharmaceutical Industry and the Medicines and Healthcare Regulatory Agency.

Tens of millions of children globally, including hundreds of thousands in the UK, have rare and life-threatening genetic diseases, with a very small number having access to a treatment. This announcement offers the prospect of change: new individualised therapeutic options are being created but there is currently no route to access these individualised medicines for most patients. The Launch Pad aims to deliver an end-to-end pathway covering diagnosis, the design and rapid manufacturing of these therapies and treatment.

 

Read the full story at the University of Oxford website.

 

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