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Brineura

This page shows the latest Brineura news and features for those working in and with pharma, biotech and healthcare.

Optimism on Orkambi in England as talks to resume

Optimism on Orkambi in England as talks to resume

And please do not forget those who desperately need #Brineura #Kuvan and #Spinraza” he tweeted.

Latest news

  • NICE knocks back BioMarin’s Batten disease drug NICE knocks back BioMarin’s Batten disease drug

    England’s cost-effectiveness watchdog has today rejected BioMarin’s Battten disease therapy Brineura (cerliponase alfa) during final guidance. ... Although not a cure, Brineura is an enzyme replacement therapy administered directly into the brain via

  • Orphan drugs dominate UK Prix Galien shortlist, medtech debuts Orphan drugs dominate UK Prix Galien shortlist, medtech debuts

    Seven companies have been shortlisted for the Orphan Product Award; Amicus (Galafold), Biogen (Spinraza), Biomarin (Brineura), Eusa (Qarziba), Intercept (Ocaliva), Kyowa Kirin (Crysvita) and Shire (Revestive).

  • CHMP backs two rare disease therapies at April meeting CHMP backs two rare disease therapies at April meeting

    Biogen's Spinraza and BioMarin's Brineura near EU approval. Two new drugs for rare diseases from Biogen and BioMarin headed the list of new therapies backed by the EMA's ... The importance of Brineura to the company has gained prominence after it was

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Latest appointments

  • Nektar Therapeutics appoints Jeff Ajer Nektar Therapeutics appoints Jeff Ajer

    officer. There he worked on establishing BioMarin’s global footprint and commercial infrastructure and has played a leadership role in the launches and growth strategies for brands including Brineura, Vimizim, Kuvan

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