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PMLiVE Top Pharma List

BioMarin

The Top Pharma List rankings are compiled from GlobalData's pharmaceutical revenue figures, which are based on prescription medicine sales, including generics drugs. They have been compiled below into an infographic of BioMarin's 2012 metabolic sales performance.

Bluebird Bio: on the cusp of a gene therapy revolution

Bluebird Bio: on the cusp of a gene therapy revolution Gilead and Novartis aside, the only companies with a stake in gene therapy with a higher market capitalisation are BioMarin, in pole position to develop a potentially curative therapy for haemophilia

Plasticell and London’s gene therapy pioneers to tackle manufacturing conundrum

Plasticell and London’s gene therapy pioneers to tackle manufacturing conundrum at UCL Medical Sciences,  and now licensed to BioMarin, which showed spectacular results in late 2017.

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).

Daily Brief: GDPR and NHS Opt Out Day, BioMarin's new rare disease approval and more

Daily Brief: GDPR and NHS Opt Out Day, BioMarin's new rare disease approval and more records. NEW RARE DISEASE APPROVAL. BioMarin sees blockbuster potential for newly-approved Palynziq. ... BioMarin has gained US approval for Palynziq (pegvaliase-pqpz) its second drug to treat adults patients with the rare condition phenylketonuria (PKU).

BioMarin ups the ante for pivotal haemophilia gene therapy trial

BioMarin ups the ante for pivotal haemophilia gene therapy trial Adds another 90 patients to the GENEr8-1 registration study. BioMarin is dramatically expanding the size of a phase III trial of its gene therapy for haemophilia A, after reporting ... There were also dramatic improvements in quality of life which

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