Children with rare inherited condition to benefit from drug through managed access agreement


11 September 2019 - It is estimated that in the UK there are around 30 to 50 children living with the condition.

A drug which slows the decline of a rare inherited condition affecting children is to be made available on the NHS.

NICE’s Highly Specialised Technology committee is supporting a positive recommendation for cerliponase alfa (Brineura, BioMarin) for children with neuronal ceroid lipofuscinosis type 2 (CLN2) – a very rare inherited condition affecting between one and six babies each year in the UK – in the context of a managed access agreement.

Read NICE press release

Michael Wonder

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Michael Wonder