Hemophilia Gene Therapy-Key Components of Clinical Care

Hemophilia Gene Therapy-Key Components of Clinical Care

Key Components of Clinical Care

Gene therapy  intends to reduce — or perhaps eliminate — the need for a regular dosage of hemophilia treatment. However, the methodology behind gene therapy is complex.

The video Hemophilia Gene Therapy - Key Components of Clinical Care is a seminar that features gene therapy researchers and education specialists in the field of hemophilia. Working with the education initiative offered by the International Society on Thrombosis and Haemostasis (ISTH), these specialists discuss the key components of clinical care associated with gene therapy for hemophilia A & B and other critical hemophilia treatment topics.

The video runs for just over 50 minutes and uses slides and commentary to discuss and explain the basics of gene therapy, including:

  • The use of viral vectors
  • The potential benefits of the single-dose treatment over traditional therapies
  • The safety and importance of clinical trials in gene therapy

Hemophilia Gene Therapy - Key Components of Clinical Care is a tailored educational study. Well-known hematologists Professor Flora Peyvandi, Professor Michael Makris, and Professor Wolfgang Miesbach of EAHAD discuss recent updates in the clinical trial process and gene therapy models for hemophilia.

This video serves as a useful preparation tool for healthcare professionals interested in earning CME credits by learning more about the potential of gene therapy as a treatment for hemophilia.

After the lecture section of the video, the team answers questions provided during the initial session. ISTH, working with global hemophilia experts, continues to develop educational resources to improve awareness of the benefits of gene therapy and the clinical application in the treatment of hemophilia. These resources provide fundamental information regarding the future of gene therapy and the latest clinical advances in the treatment of hemophilia.

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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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