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Publications

Learn more about Hemophilia A and BioMarin therapies

Featured Publications:

Hemophilia A Publications List:

Publication

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  • Publications
  • Hemophilia A
  • Real World Evidence

Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance

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Publication

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  • Publications
  • Hemophilia A
  • Safety and Efficacy Profile

Estimated long-term durability of valoctocogene roxaparvovec treatment in male patients with severe hemophilia a: An extrapolation of clinical data

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Publication

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  • Publications
  • Hemophilia A
  • Safety and Efficacy Profile

Comparative effectiveness of valoctocogene roxaparvovec and efanesoctocog alfa in the treatment of severe hemophilia a: A matching-adjusted indirect comparison of bleeding frequency

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Plain Language Summaries

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  • Publications
  • Hemophilia A
  • Safety and Efficacy Profile

Plain language summary of 4-year results of the GENEr8-1 clinical trial of valoctocogene roxaparvovec gene therapy for hemophilia a

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Plain Language Summaries

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  • Publications
  • Hemophilia A
  • Safety and Efficacy Profile

A plain language summary on indirectly comparing bleeding after valoctocogene roxaparvovec gene therapy to bleeding with emicizumab prophylaxis

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Plain Language Summaries

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  • Plain Language Summaries
  • Hemophilia A

Plain language summary of the GENEr8-1 clinical trial of valoctocogene roxaparvovec gene therapy for hemophilia A

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Selected Publications

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  • Publications
  • Hemophilia A

Access selected clinical, pre-clinical and other publications for Hemophilia A

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