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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 and Glenn F. Peirce, MD, PhD
- 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
- Created on 16 October 2019