Seven-year follow-up of valoctocogene roxaparvovec gene therapy for haemophilia A

Highlights From the 17th Annual Congress of EAHAD

Human liver biopsy analysis reveals lower RNA transcription may contribute to a decline in FVIII levels following AAV5-hFVIII-SQ gene therapy

Highlights From the 17th Annual Congress of EAHAD

Stable and durable factor IX (FIX) levels over 4 years after etranacogene dezaparvovec gene therapy administration in a Phase 2b trial in patients with haemophilia B

Highlights From the 17th Annual Congress of EAHAD

Health-related Quality of Life in Adults With Hemophilia B After Gene Therapy With Fidanacogene Elaparvovec in the BENEGENE-2 Trial

Highlights From the 17th Annual Congress of EAHAD

Safety and efficacy of valoctocogene roxaparvovec in participants with active and prior FVIII inhibitors

Highlights From the 17th Annual Congress of EAHAD

A Novel Gene Editing Lexicon Strategy for the Haemophilia Community

Highlights From the 17th Annual Congress of EAHAD

A New Tool to Assist in Treatment Selection–The World Federation of Hemophilia (WFH) Shared Decision-making (SDM) Tool

Highlights From the 17th Annual Congress of EAHAD

Health-related Quality of Life in Adults With Hemophilia B After Receiving Gene Therapy With Fidanacogene Elaparvovec

Highlights From the 65th ASH Annual Meeting and Exposition

HIV Comorbid Infection and Liver-directed AAV-Based Gene Therapy in Adults With Severe and Moderately Severe Hemophilia B

Highlights From the 65th ASH Annual Meeting and Exposition

Long-term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption, and Safety of Hemophilia B Gene Therapy

Highlights From the 65th ASH Annual Meeting and Exposition

Development of adeno-associated virus (AAV5) variants capable of reducing the immunogenicity by targeting antigenic residues on the capsid surface

Highlights From the ESGCT 30th Annual Congress

Phase 3 HOPE-B Trial of etranacogene dezaparvovec in Severe/Moderately Severe Hemophilia B

Highlights From the 31st Congress of ISTH

Updates on SPK-8011

Highlights From the 31st Congress of ISTH

Updates from the GENEr8-1 Trial

Highlights From the 31st Congress of ISTH

Residual Burden of Liver Disease After HCV-clearance in Hemophilia: A Word of Caution in the Era of Gene Therapy

Highlights From the 31st Congress of ISTH

Efficacy and Safety of Fidanacogene Elaparvovec in Adults With Moderately Severe or Severe Hemophilia B: Results From the Phase 3 BENEGENE-2 Gene Therapy Trial

Highlights From the 31st Congress of ISTH

Elucidating the Mechanism Behind the AAV-Derived Factor VIII Assay Discrepancy

Conference Coverage

Relationship Between Transgene-Produced FVIII and Bleeding Rates 2 Years After Gene Transfer With Valoctocogene Roxaparvovec: Results From Gener8-1

Conference Coverage

Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients With Hemophilia B

Conference Coverage

Stable Hemostatic Correction and Improved Hemophilia-Related Quality of Life: Final Analysis from the Pivotal Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec

Conference Coverage

A Factor VIII Variant With Elimination of the N-glycosylation Site at 2118 Reduced Anti-FVIII Immune Responses in Gene Therapy Treated Hemophilia A Mice

Conference Coverage

Stabilin-2 Promoter Modulates the Immune Response to FVIII After Lentiviral Vector Delivery in Hemophilic Mice

Conference Coverage

Delivery of CRISPR/Cas9 mRNA LNPs to Repair a Small Deletion in FVIII Gene in Hemophilia A Mice

Conference Coverage

Using the World Federation of Haemophilia Gene Therapy Registry for the Long-term Follow-up of Haemophilia Patients Treated with Gene Therapy

Conference Coverage

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Highlights From the 17th Annual Congress of EAHAD

Highlights From the 65th ASH Annual Meeting and Exposition

Highlights From the ESGCT 30th Annual Congress

Highlights From the 31st Congress of ISTH

Highlights From the 32nd Congress of ISTH

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Supported through educational grants from BioMarin, CSL Behring, Pfizer Inc., Roche, and Spark Therapeutics

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