BioMarin Archives - Pharmaphorum

Gene therapy firm BioMarin stages haemophilia musical

https://pharmaphorum.com/news/gene-therapy-firm-biomarin-stages-haemophilia-musical/

FDA approves new treatment for rare genetic disease PKU

https://pharmaphorum.com/news/fda-approves-new-treatment-for-rare-genetic-disease-pku/

Nektar, BioMarin, and Alnylam top list of biotech M&A targets

https://pharmaphorum.com/news/nektar-biomarin-and-alnylam-top-list-of-biotech-ma-targets/

NICE rejects Biomarin’s £520K per year rare disease drug

https://pharmaphorum.com/news/nice-rejects-biomarins-520/

‘Cure for hemophilia within reach’ as BioMarin and Spark gene therapies shine

https://pharmaphorum.com/news/cure-hemophilia-within-reach-biomarin-spark-gene-therapies-shine/

Breakthrough status for BioMarin ratchets up haemophilia A competition

https://pharmaphorum.com/news/breakthrough-status-biomarin-ratchets-haemophilia-competition/

EU regulators back two more orphan drugs, question of pricing remains

https://pharmaphorum.com/news/eu-regulators-back-two-orphan-drugs-amid-concerns-price/

New NICE rules could deny drugs to rare disease patients

https://pharmaphorum.com/news/england-rare-disease-broken/

Rare disease drug Vimizim gets NICE ‘yes’ – but needs real-world data

https://pharmaphorum.com/views-and-analysis/rare-disease-drug-vimizim-gets-nice-yes/

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