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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
February 23, 2019NICE has published draft guidance which confirms its earlier decision not to recommend cerliponase alfa (also called Brineura and made by Biomarin) for children with Batten disease - a very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
Pharma Sources Insight January 2025
