Vimizim

News

NICE firms up NHS use of Vimizim for Morquio A syndrome

In 2015, NICE said that BioMarin's Vimizim could be used to treat patients with the ultra-rare disease Morquio A syndrome, while real-world data was collected on its efficacy.

News

Rare disease drug Strensiq too expensive, says NICE

Alexion's new rare disease treatment Strensiq has been rejected by England's health watchdog NICE in draft guidance.

Articles

Rare disease drug Vimizim gets NICE 'yes' – but needs real-w...

NICE says it can approve BioMarin's ultra-rare drug treatment Vimizim, but has proposed real-world data collection and a price cap to overcome doubts about the drug's value, reports Andrew McCo

NICE firms up NHS use of Vimizim for Morquio A syndrome

Rare disease drug Strensiq too expensive, says NICE

Rare disease drug Vimizim gets NICE 'yes' – but needs real-w...

Speech analysis tech for ALS gets FDA breakthrough tag

Trials and tribulations of drug development

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