Page 13 - HFA Dateline 2022 Special Edition
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                            What we know—and don’t know—

                               about emerging gene therapies

                                        BY DARA CHADWICK, FREELANCE WRITER

                             magine a day when you or someone you love no longer needs to spend
                             time each week getting prophylactic treatment for a bleeding disorder.
                          IGone is the time spent driving to a treatment center. Gone is the need to
                           sit through scheduled time-consuming intravenous (IV) factor infusions. Gone,
                           too, is the fear of repeated bleeds and their long-term health impacts.

                           This is the potential of gene therapy. And while this potential has yet to be
                           fully realized, experts in bleeding disorders treatment say an approved gene
                           therapy is well within our grasp.

                           “Gene therapy has the potential to make a huge difference,” said David Clark,
                           PhD, chair of the Coalition for Hemophilia B. Clark is a retired pharmaceutical
                           scientist who helped develop plasma and recombinant factor VIII (FVIII) and
                           factor IX (FIX) products.

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