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1. Intermediate: (Tags); Intermediate; Created on 28 September 2021
2. A History of Hemophilia Treatment: Factor Replacement to Gene Therapy: (Article tagged with: Intermediate); Presented by Flora Peyvandi, MD, PhD; Created on 30 June 2019
3. A Novel Adeno Associated Virus (AAV) Gene Therapy (FLT180a) Achieves Normal FIX Activity Levels in Severe Hemophilia B (HB) Patients (B-AMAZE Study): (Article tagged with: Intermediate); Pratima Chowdary, MRCP, FRCPath; Created on 21 August 2020
4. Adeno-Associated Virus (AAV) Mediated Gene Transfer: Why is This the Predominant Strategy for Hemophilia Gene Therapy?: (Article tagged with: Intermediate); Presented by Steven W. Pipe, MD and Nigel Key, MB, ChB. FRCP; Created on 05 December 2019
5. 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: (Article tagged with: Intermediate); Wolfgang A. Miesbach, MD; Created on 05 December 2020
6. 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: (Article tagged with: Intermediate); Frank W.G. Leebeek, MD, PhD; Created on 04 March 2021
7. EAHAD Gene Therapy Working Group: (Article tagged with: Intermediate); Presented by Wolfgang Miesbach, MD; Created on 21 December 2020
8. 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: (Article tagged with: Intermediate); Steven W. Pipe, MD; Created on 03 March 2021
9. 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: (Article tagged with: Intermediate); Steven W. Pipe, MD; Created on 05 December 2020
10. 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 ...: (Article tagged with: Intermediate); Steven W. Pipe, MD; Created on 05 December 2020
11. First-in-human Four-year Follow-up Study of Durable Therapeutic Efficacy and Safety: AAV Gene Therapy With Valoctocogene Roxaparvovec for Severe Haemophilia A: (Article tagged with: Intermediate); John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH; Created on 10 July 2020
12. 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: (Article tagged with: Intermediate); Steven W. Pipe, MD; Created on 05 December 2020
13. First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A: Safety and FVIII Activity Results: (Article tagged with: Intermediate); Steven W. Pipe, MD; Created on 29 June 2020
14. 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): (Article tagged with: Intermediate); Pratima Chowdary, MRCP, FRCPath; Created on 04 March 2021
15. Gene Therapy for Treatment of Hemophilia: An Introduction to Adeno-Associated Viral Vector Gene Transfer: (Article tagged with: Intermediate); Presented by Johnny Mahlangu, BSc, MBBCh, MMed, FCPath; Created on 05 December 2019
16. Gene Therapy for Treatment of Hemophilia: Common Concerns in Gene Therapy: (Article tagged with: Intermediate); Presented by Thierry VandenDriessche, PhD; Created on 25 November 2019
17. Gene Therapy for Treatment of Hemophilia: Other Strategies and Targets: (Article tagged with: Intermediate); Presented by Glenn F. Pierce, MD, PhD; Created on 10 September 2019
18. Gene Therapy Illustrated Clinical Trials: (Article tagged with: Intermediate); Created on 29 September 2021
19. Highlights from the NHF 15th Workshop on Novel Technologies and Gene Transfer for Hemophilia: (Article tagged with: Intermediate); Presented by David Lillicrap, MD; Created on 16 October 2019
20. Highlights from the NHF 15th Workshop on Novel Technologies and Gene Transfer for Hemophilia: (Article tagged with: Intermediate); Presented by David Lillicrap, MD and Glenn F. Peirce, MD, PhD; Created on 16 October 2019

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Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

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