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Home
About
Roadmap
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About ISTH
Blog
Topics
Basics of Hemophilia
Fundamental of Gene Therapies
Practical Considerations of Gene Therapies
Clinical Trial Efficacy and Safety
Patient Journey: Implementation to Lived Experience
Clinical Perspectives/Real-World Evidence
Roles of the Multidisciplinary Team
Recent Updates and Ongoing Efforts
Education
Resources Builder
Shared Decision Making
HTC Case Studies
EXPLORE Modules
Illustrated Clinical Trials
Interactive Webinars
Podcasts
Roadmap
Gene Therapy Resources
Conference Coverage
Microlearning
Monthly Roundup
News
Learning Tiers
Introductory
Intermediate
Advanced
Home
About
Roadmap
Steering Committee
About ISTH
Blog
Topics
Basics of Hemophilia
Fundamental of Gene Therapies
Practical Considerations of Gene Therapies
Clinical Trial Efficacy and Safety
Patient Journey: Implementation to Lived Experience
Clinical Perspectives/Real-World Evidence
Roles of the Multidisciplinary Team
Recent Updates and Ongoing Efforts
Education
Resources Builder
Shared Decision Making
HTC Case Studies
EXPLORE Modules
Illustrated Clinical Trials
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Roadmap
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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
30 July 2024
Health-related Quality-of-Life Outcomes 4 Years After Treatment with Valoctocogene Roxaparvovec
24 July 2024
Efficacy and Safety of Valoctocogene Roxaparvovec 4 Years After Gene Transfer in GENEr8-1
27 June 2024
Seven-year follow-up of valoctocogene roxaparvovec gene therapy for haemophilia A
10 March 2024
Human liver biopsy analysis reveals lower RNA transcription may contribute to a decline in FVIII levels following AAV5-hFVIII-SQ gene therapy
10 March 2024
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
10 March 2024
Health-related Quality of Life in Adults With Hemophilia B After Gene Therapy With Fidanacogene Elaparvovec in the BENEGENE-2 Trial
04 March 2024
Safety and efficacy of valoctocogene roxaparvovec in participants with active and prior FVIII inhibitors
01 March 2024
A Novel Gene Editing Lexicon Strategy for the Haemophilia Community
12 February 2024
A New Tool to Assist in Treatment Selection–The World Federation of Hemophilia (WFH) Shared Decision-making (SDM) Tool
11 February 2024
Health-related Quality of Life in Adults With Hemophilia B After Receiving Gene Therapy With Fidanacogene Elaparvovec
11 January 2024
Bleeding Outcomes in Participants With Factor VIII Activity
19 December 2023
Long-term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption, and Safety of Hemophilia B Gene Therapy
19 December 2023
HIV Comorbid Infection and Liver-directed AAV-Based Gene Therapy in Adults With Severe and Moderately Severe Hemophilia B
19 December 2023
Development of adeno-associated virus (AAV5) variants capable of reducing the immunogenicity by targeting antigenic residues on the capsid surface
18 December 2023
Updates from the GENEr8-1 Trial
31 July 2023
Subcategories
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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Home
About
Roadmap
Steering Committee
About ISTH
Blog
Topics
Basics of Hemophilia
Fundamental of Gene Therapies
Practical Considerations of Gene Therapies
Clinical Trial Efficacy and Safety
Patient Journey: Implementation to Lived Experience
Clinical Perspectives/Real-World Evidence
Roles of the Multidisciplinary Team
Recent Updates and Ongoing Efforts
Education
Resources Builder
Shared Decision Making
HTC Case Studies
EXPLORE Modules
Illustrated Clinical Trials
Interactive Webinars
Podcasts
Roadmap
Gene Therapy Resources
Conference Coverage
Microlearning
Monthly Roundup
News
Learning Tiers
Introductory
Intermediate
Advanced
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