Remove tag haemophilia-b
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Can gene therapies for haemophilia defend their high price tags?

Pharmaceutical Technology

Despite their potential multi-million-dollar sticker price, if haemophilia treatments by BioMarin and CSL Behring prove durable, they could help patients save money compared to current treatments, experts note. A fair price for CSL Behring’s haemophilia B treatment etranacogene dezaparvovec would be between $2.93–2.96

Safety 111
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CSL doses first patient with haemophilia B gene therapy Hemgenix

Pharmaceutical Technology

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. This followed positive results from CSL’s Phase III HOPE-B trial (NCT03569891).