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Effectiveness concerns for drug used in Batten disease

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Effectiveness concerns for drug used in Batten disease

NICE has published draft guidance confirming that it will not recommend cerliponase alfa (also called Brineura and made by Biomarin) for children with Batten disease – a very rare inherited condition affecting between one and six babies each year in the UK.

 

The decision is based on concerns about the long-term effectiveness of the drug.