First Approval of Gene Therapy in Hemophilia

First Approval of Gene Therapy in Hemophilia

Steven W. Pipe, MD comments on the recent approval.

First Approval of Gene Therapy in Hemophilia

Highlights from the 15th Annual Congress of EAHAD

Highlights from the 15th Annual Congress of EAHAD

EXPLORE™ Module

EXPLORE™ Module

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Train-the-Trainer Toolkit

Train-the-Trainer Toolkit

Prepare for the future approval of a gene therapy in hemophilia

Interactive Webinar

Interactive Webinar

Obstacles and Opportunities
Presented by: Frank W.G. Leebeek, MD, PhD

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Interactive Webinar

Conference Coverage

Updates on Three Ongoing Clinical Trials on Adults with Severe Hemophilia B

Conference Coverage

Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People With Hemophilia A

Conference Coverage

Relationship Between Transgene-Produced FVIII and Bleeding Rates 2 Years After Gene Transfer With Valoctocogene Roxaparvovec: Results From Gener8-1

Conference Coverage

Elucidating the Mechanism Behind the AAV-Derived Factor VIII Assay Discrepancy

Latest News

BioMarin Announces Stable and Durable Annualized Bleed Control for ROCTAVIAN™️ in Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A

08 January 2023

Pfizer Announces Positive Top-Line Results from Phase 3 Study of Hemophilia B Gene Therapy Candidate

29 December 2022

BioMarin Says FDA No Longer Plans Advisory Meeting for Roctavian

24 November 2022

FDA Approves First Gene Therapy to Treat Adults with Hemophilia B

23 November 2022

First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN™ Approved by European Commission (EC)

24 August 2022

Conference Coverage

Highlights From the 30th Congress of ISTH

CME INFORMATION

Elucidating the Mechanism Behind the AAV-Derived Factor VIII Assay Discrepancy

Elucidating the Mechanism Behind the AAV-Derived Factor VIII Assay Discrepancy

Anna Sternberg, PhD

Relationship Between Transgene-Produced FVIII and Bleeding Rates 2 Years After Gene Transfer With Valoctocogene Roxaparvovec: Results From Gener8-1

Relationship Between Transgene-Produced FVIII and Bleeding Rates 2 Years After Gene Transfer With Valoctocogene Roxaparvovec: Results From Gener8-1

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath

Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients With Hemophilia B

Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients With Hemophilia B

Guy Young, MD

Improvements in Health-Related Quality of Life in Adults With Severe or Moderately Severe Hemophilia B After Receiving Etranacogene Dezaparvovec Gene Therapy

Improvements in Health-Related Quality of Life in Adults With Severe or Moderately Severe Hemophilia B After Receiving Etranacogene Dezaparvovec Gene Therapy

Steven W. Pipe, M

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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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