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Authors
Santos, Sandra
Robinson, Tara M.
Trueman, David
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Clinical Trials BETA
Clinical trials referenced in this document:
nct03370913 at ClinicalTrials.govnct02576795 at ClinicalTrials.govDocuments that mention this clinical trial
Estimated Long-Term Durability of Valoctocogene Roxaparvovec Treatment in Male patients with Severe Hemophilia A: An Extrapolation of Clinical Data
https://doi.org/10.1007/s12325-025-03368-4
Comparative Effectiveness of Valoctocogene Roxaparvovec and Prophylactic Factor VIII Replacement in Severe Hemophilia A
https://doi.org/10.1007/s12325-024-02834-9
Documents that mention this clinical trial
Estimated Long-Term Durability of Valoctocogene Roxaparvovec Treatment in Male patients with Severe Hemophilia A: An Extrapolation of Clinical Data
https://doi.org/10.1007/s12325-025-03368-4
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Funding
Funding for this research was provided by:
BioMarin Pharmaceutical
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Article History
Received: 18 June 2025
Accepted: 2 September 2025
First Online: 23 September 2025
Declarations
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: David Trueman is an employee of Source Health Economics, who were funded by BioMarin to undertake this work. Sandra Santos is an employee and stockholder of BioMarin UK Ltd. Tara Robinson was an employee and stockholder of BioMarin Pharmaceutical Inc at the time of study initiation.
: This study is a post hoc analysis of previously conducted studies and does not include the addition of new studies with human participants or animal subjects. As previously reported, the protocols for GENEr8-1 [] and Study 270–201 [] were approved by the institutional review boards or independent ethics committees of all participating sites, and all participants provided written informed consent to participate. Both trials were performed in accordance with the ethical principles set forth by the Declaration of Helsinki.
