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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
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
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Roadmap
Gene Therapy Resources
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Monthly Roundup
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WHAT ARE YOU LOOKING FOR?
Intermediate
Follow-up on a Novel Adeno-Associated Virus (AAV) Gene Therapy (FLT180a) Achieving Normal FIX Activity Levels in Severe Hemophilia B (HB) Patients (B-AMAZE Study)
04 March 2021
Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: First Data From the Phase 3 Hope-B Gene Therapy Trial
03 March 2021
Long-term Outcomes: Durability and Safety
28 January 2021
Key Considerations: Advances in Gene Therapy for Hemophilia ENDURING
22 December 2020
EAHAD Gene Therapy Working Group
21 December 2020
Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults With Severe Hemophilia
05 December 2020
The World Federation of Hemophilia Gene Therapy Registry
05 December 2020
AMT-060 Gene Therapy in Adults With Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for Up to 5 Years
05 December 2020
First Data From the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults With Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing
05 December 2020
Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B: Two Year Data From a Phase 2b Trial
05 December 2020
First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A – BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII
05 December 2020
Latest Advances in Gene Therapy for Hemophilia in Portuguese
23 November 2020
Obstacles and Opportunities
20 November 2020
Stepping Into the Future: Hemophilia Gene Therapy Clinical Trial Updates
25 September 2020
Patient Support, Patient Counseling, and Monitoring
23 September 2020
Learning from Clinical Trials and Preparing for Clinical Practice
22 September 2020
Monthly Roundup
Interactive Webinars
Podcasts
Conference Coverage
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
Gene Therapy Illustrated Clinical Trials
Microlearning
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Supported through educational grants from BioMarin, CSL Behring, Pfizer Inc., Roche, and Spark Therapeutics
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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