Glenn Pierce and Steve Pipe discuss the latest updates on Gene Therapy for hemophilia | July 2021


Year: 2021

Language: English

Author(s): World Federation of Hemophilia

Also Available In: Spanish, French, Arabic, Russian

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Additional Information

Listen to Glenn Pierce, MD, PhD and Steve Pipe, MD discuss the latest gene therapy information that was presented at the ISTH 2021 Congress.

To skip to a specific question, please use the timestamps below:

  • 01:25 Variability of expression between patients
  • 03:00 Range of expression
  • 03:40 Durability of expression
  • 07:05 Can we predict which patients will do well on gene therapy, and which won’t?
  • 10:44 Correlation between expression levels and bleed protection
  • 12:48 Short-term safety
  • 14:50 Long-term safety
  • 16:39 HOPE-B hepatocellular carcinoma case

The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. For diagnosis or consultation on a specific medical problem, the WFH recommends that you contact your physician or local treatment centre. Before administering any products, the WFH urges patients to check dosages with a physician or hemophilia centre staff, and to consult the pharmaceutical company’s printed instructions.

The WFH does not promote any particular pharmaceutical product and any mention of any commercial brand in this presentation is strictly for educational purposes.