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Brineura
This page shows the latest Brineura news and features for those working in and with pharma, biotech and healthcare.
NICE approves BioMarin’s Brineura after initial setback
The positive recommendation of Brineura was agreed by NICE’s Highly Specialised Technology committee. ... Although Brineura does not does not cure the disease, NICE agreed that it is an important development in the treatment of the condition.
Latest news
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BioMarin ticks over in Q2 ahead of gene therapy filing
our late stage clinical programmes, and higher marketing and sales expenses in support of the commercial launches of Palynziq and Brineura”. ... BioMarin will also hope to extend the commercial success of Brineura, its Batten disease therapy.
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Optimism on Orkambi in England as talks to resume
And please do not forget those who desperately need #Brineura #Kuvan and #Spinraza” he tweeted.
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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
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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).
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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 Intelligence
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Rare disease drug development – real-world evidence innovations with historical controls
Examples of historical control applications. Historical control information from an ongoing patient registry was used in the approval of Brineura (cerliponase alfa), the enzyme replacement therapy that helps treat CLN2 disease, ... Brineura was approved
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Latest appointments
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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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