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Gene Therapy in Hemophilia

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Conference Coverage

Improved hFIX and hFVIII expression with ITR-modified AAV vectors in hemophilic mice

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Evolution Directed Gene Engineered (EDGE) Gain-of-Function Factor VIII Variants Enhance Gene Therapy for Hemophilia A

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Durable Bleed Protection for All Bleeding Subtypes and Favorable Safety Over 5 Years (end-of-study) in the Phase 3 HOPE-B Trial for Persons With Severe or Moderately Severe Haemophilia B

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Stable Factor IX Expression and Sustained Reductions in Factor IX Use 9 Years After Gene Therapy With CSL220 in Adults With Haemophilia B

Latest News

Role of the Nurse Coordinator in Hemophilia Gene Therapy

26 July 2026

An analysis of attitudes toward gene therapy in people with severe hemophilia in Germany, a survey-based cross-sectional study

02 June 2026

Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: Final phase 3 GENEr8-1 trial results

27 March 2026

Building a gene editing lexicon: a model for rare and inherited disorders

13 March 2026

An analysis of attitudes toward gene therapy in people with severe hemophilia in Germany, a survey-based cross-sectional study

11 March 2026

CONFERENCE COVERAGE

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

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

30 July 2024Highlights From the 32nd Congress of ISTH

Adam Cuker, MD, MS

Health-related Quality-of-Life Outcomes 4 Years After Treatment with Valoctocogene Roxaparvovec

Health-related Quality-of-Life Outcomes 4 Years After Treatment with Valoctocogene Roxaparvovec

24 July 2024Highlights From the 32nd Congress of ISTH

Bella Madan, MD, FRCP, FRCPath

Efficacy and Safety of Valoctocogene Roxaparvovec 4 Years After Gene Transfer in GENEr8-1

Efficacy and Safety of Valoctocogene Roxaparvovec 4 Years After Gene Transfer in GENEr8-1

27 June 2024Highlights From the 32nd Congress of ISTH

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath

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

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