Year: 2020
Language: English
Author(s): World Federation of Hemophilia
In this session from the WFH 2020 World Summit, Glenn Pierce will examine the evolution of gene therapy while addressing the unknowns that need to be answered.
Hemophilia is at the dawn of a new era in therapeutic management, one that can generate greater protection from bleeding and a functional cure in some individuals. The use of gene transfer is emerging as an effective long-term treatment for a variety of diseases. While clinical progress has been definitive, many questions remain unanswered as pre-licensure phase 3 clinical trials are underway. These unanswered questions translate into a state of uncertainty about the known unknowns and unknown unknowns intrinsic to any new therapeutic platform.
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.
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