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- PMLiVE

BioMarin

Biomarin

BioMarin drug boosts growth in children with dwarfism

Vosoritide showed results in 50% of trial participants

Biomarin

BioMarin files muscular dystrophy drug in EU

Drisapersen could be the first ‘exon-skipping’ therapy

- PMLiVE

Baxter plans to file long-acting Advate in EU next year

Patients experienced a 95% reduction in bleeds

- PMLiVE

FDA approves Baxter drug for rare form of haemophilia

Obizur is derived from pig proteins

- PMLiVE

Baxter ‘will file long-acting haemophilia drug by year-end’

New version of Advate clears late-stage trial

Sanofi reception

Sanofi and Regeneron pay $67.5m to speed alirocumab review

Buy FDA priority review voucher from BioMarin

- PMLiVE

FDA approves Biogen Idec’s haemophilia drug

Eloctate now available to help control and prevent bleeding episodes

Biomarin

EC approves Biomarin rare genetic disorder drug

Vimizim now available to treat Morquio A syndrome

- PMLiVE

FDA backs BioMarin drug for rare enzyme disorder

Vimizim is first drug approved in US under rare paediatric disease priority review

- PMLiVE

Boehringer backs gene therapy firm Eyevensys

Invests in French eye disease specialist

- PMLiVE

Basilea’s Hans Christian Rohde takes up commercial role at uniQure

Will serve as gene therapy specialist's chief commercial officer

- PMLiVE

Gene therapy: the promise comes closer

After decades of development and a trail of failed clinical trials, gene therapy is finally emerging as a worthwhile approach for treating a range of acquired and inherited diseases

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