drug
valoctocogene roxaparvovec
Marketedupdated 3mo agoby $BMRN BIOMARIN PHARMACEUTICAL INC
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Trials studying valoctocogene roxaparvovec
- phase2Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
- phase3Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
- phase3Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
- phase2Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
- phase2Gene Therapy Study in Severe Haemophilia A Patients (270-201)
- phase3Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
- phase3Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
News(1)
- BioMarin axes asset from $270M Inozyme takeover, ending run in rare metabolic indicationBioMarin’s discontinued drug showed biomarker benefits in a Phase 3 study for ENPP1 deficiency but these failed to translate to clinical improvements.biospace · 4d ago
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