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Education

Preparing Treatment Centers for Administration

Podcasts

Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Wolfgang A. Miesbach, MD, PhD

Patient Perspectives for Hemophilia in Gene Therapy

Podcasts

Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Declan Noone

Gene Therapy for Hemophilia A and B Compared to Available Treatments

Podcasts

Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Maria Elisa Mancuso, MD, PhD

Gene Therapy in Hemophilia Ongoing and Future Research and What to Expect

Podcasts

Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Dr. Steven W. Pipe, MD

Importance of Monitoring Individuals With Hemophilia for Safety and Efficacy in the Short Term and Long Term

Podcasts

Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Pratima Chowdary, MRCP, FRCPath

Monitoring of Vector Integration Sites in In Vivo Gene Therapy Approaches by Liquid-Biopsy-Integration-Site Sequencing

Conference Coverage

Daniela Cesana, PhD

Overcoming anti-AAV pre-existing immunity to achieve safe and efficient gene transfer in clinical settings

Conference Coverage

Giuseppe Ronzitti, PhD

Lentiviral Gene Therapy to the Liver for Hemophilia and Beyond

Conference Coverage

Alessio Cantore, PhD

Current Update of Clinical trials on Gene Therapy for Hemophilia

Conference Coverage

Wolfgang Miesbach, MD

Preparing for Practice Readiness in Gene Therapy for Hemophilia

Virtual Learning Sessions

Wolfgang Miesbach, MD and Steven W. Pipe, MD

Updates in Clinical Trials in Gene Therapy for Hemophilia A

Podcasts

Presented by Presented by Margareth C. Ozelo, MD, PhD

Latest Clinical Trial Updates for Hemophilia B Gene Therapy

Podcasts

Presented by Margareth C. Ozelo, MD, PhD

Recent Advances in Gene Therapy for Hemophilia Clinical Trials

Virtual Learning Sessions

Presented by Annette von Drygalski, MD, PharmD and Guy Young, MD

ISTH Gene Therapy in Hemophilia Train-the-Trainer

Toolkit

Expert of the Month Interviews

Expert of the Month Interviews

Gene Therapy Illustrated Clinical Trials

Gene Therapy Illustrated Clinical Trials

Clinical Outcomes in Adults With Hemophilia B With and Without Pre-existing Neutralizing Antibodies to AAV5: 6 Month Data From the Phase 3 Etranacogene Dezaparvovec HOPE-B Gene Therapy Trial

Conference Coverage

Wolfgang A. Miesbach, MD, PhD

Efficacy and Safety of Valoctocogene Roxaparvovec Adeno-associated Virus Gene Transfer for Severe Hemophilia A: Results From the Phase 3 GENEr8-1 Trial

Conference Coverage

Margareth C. Ozelo, MD, PhD

Liver Safety Case Report From the Phase 3 HOPE-B Gene Therapy Trial in Adults With Hemophilia B

Conference Coverage

Steven W. Pipe, MD

Investigation of Early Outcomes Following Adeno-associated Viral Gene Therapy in a Canine Hemophilia Model

Conference Coverage

David Lillicrap, MD

Evolution of AAV Vector Gene Therapy Is Ongoing in Hemophilia. Will the Unique Features of BAY 2599023 Address the Outstanding Needs?

Conference Coverage

Steven W. Pipe, MD

52 Week Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: Data From the Phase 3 HOPE-B Gene Therapy Trial

Conference Coverage

Steven W. Pipe, MD

Train-the-Trainer Toolkit

Toolkit

Gene Therapy in Hemophilia: An APSTH ISTH Sponsored Symposium

Virtual Learning Sessions

Presented by Pantep Angchaisuksiri, MD, Flora Peyvandi, MD, PhD, Steven W. Pipe, MD, and Alok Srivastava, MD, FRACP, FRCPA, FRCP

Subcategories

Interactive Webinars

Expert of the Month Interviews

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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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