Morquio

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.

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

News

Neurogene drops high-dose arm in Rett study after death

The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died

Sales & Marketing

Coming soon: The Life Sciences Industry Report 2025 – insigh...

Prepare for 2025 with pharmaphorum’s Life Sciences Report, featuring expert insights on AI in drug development, gene therapy, and top industry trends

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