Efficacy and Safety of an Engineered, Liver-Tropic Adeno-Associated Virus Vector Expressing Factor IX Padua Administered with Prophylactic Glucocorticoids in Patients with Hemophilia B: A Multi-Center, Single-Arm, Phase 3 Trial
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Efficacy and Safety of an Engineered, Liver-Tropic Adeno-Associated Virus Vector Expressing Factor IX Padua Administered with Prophylactic Glucocorticoids in Patients with Hemophilia B: A Multi-Center, Single-Arm, Phase 3 Trial
Gene Therapy for Sickle Cell Disease Is Associated with a Decreased Transplant Length of Stay and Similar Rates of Short-Term Complications Compared to Allogeneic Matched Sibling Stem Cell Transplant: A Single Center Experience
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Gene Therapy for Sickle Cell Disease Is Associated with a Decreased Transplant Length of Stay and Similar Rates of Short-Term Complications Compared to Allogeneic Matched Sibling Stem Cell Transplant: A Single Center Experience
Improved Joint Health in the Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B over 36 Months of Follow up
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Improved Joint Health in the Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B over 36 Months of Follow up
In Vitro and In Vivo Potency Differences between AAVRh74var and AAV5 Vectors Encoding the Same High-Activity Human Factor IX Variant, FIX-R338L, Expression Cassette: Implications for Hemophilia B Gene Therapy
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In Vitro and In Vivo Potency Differences between AAVRh74var and AAV5 Vectors Encoding the Same High-Activity Human Factor IX Variant, FIX-R338L, Expression Cassette: Implications for Hemophilia B Gene Therapy
Participants with a History of Stroke in Lovotibeglogene Autotemcel (Lovo-cel) Clinical Trials
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Participants with a History of Stroke in Lovotibeglogene Autotemcel (Lovo-cel) Clinical Trials
Prophylactic Administration of Glucocorticoids and Tacrolimus in AAV8-F8 Gene Therapy of Severe Haemophilia-a Achieved a Significant Long-Term Efficacy
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Prophylactic Administration of Glucocorticoids and Tacrolimus in AAV8-F8 Gene Therapy of Severe Haemophilia-a Achieved a Significant Long-Term Efficacy
RP-L301, a Lentiviral-Mediated Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase 2 Clinical Trial Design
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RP-L301, a Lentiviral-Mediated Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase 2 Clinical Trial Design
Results from the Hemophilia Α Cohort of the Non-Investigational Lead-in Study: Prospective Collection of Bleeding Rate in Participants with Hemophilia Α Prior to Phase 3 Study (AFFINE) of Giroctocogene Fitelparvovec
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Results from the Hemophilia Α Cohort of the Non-Investigational Lead-in Study: Prospective Collection of Bleeding Rate in Participants with Hemophilia Α Prior to Phase 3 Study (AFFINE) of Giroctocogene Fitelparvovec
Stable Factor IX Expression and Sustained Reductions in Factor IX Use 8 Years after Gene Therapy with CSL220 (Formerly AMT-060) in Adults with Hemophilia B
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Stable Factor IX Expression and Sustained Reductions in Factor IX Use 8 Years after Gene Therapy with CSL220 (Formerly AMT-060) in Adults with Hemophilia B
Use of Fidanacogene Elaparvovec, a Gene Therapy Vector, to Deliver a Stable, Fully Functional Human Factor IX Transgene for the Treatment of Hemophilia B: A Combined Analysis of Safety
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Use of Fidanacogene Elaparvovec, a Gene Therapy Vector, to Deliver a Stable, Fully Functional Human Factor IX Transgene for the Treatment of Hemophilia B: A Combined Analysis of Safety