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BioMarin seeks haemophilia gene therapy approval after promising data
https://pharmaphorum.com/news/biomarin-seeks-haemophilia-gene-therapy-approval-after-promising-data/
BioMarin
FDA approves new treatment for rare genetic disease PKU
Novel enzyme therapy will treat adults with PKU who have uncontrolled blood phenylalanine concentrations with current treatment.
NICE rejects Biomarin’s £520K per year rare disease drug
Company will negotiate access scheme
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