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You are here: Industry Insights > long-term effectiveness

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NICE says drug to treat Batten disease cannot be recommended for NHS use because company is unable to address concerns about long-term effectiveness firstwordpharma
February 23, 2019
NICE has published draft guidance which confirms its earlier decision not to recommend cerliponase alfa (also called Brineura and made by Biomarin) for children with Batten disease - a very rare inherited condition affecting between 1 and 6 babies each ye

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