The WFH is proud to announce that we will be collaborating with St. Jude Children’s Research Hospital in order to establish a gene therapy clinical trial for hemophilia patients in low- and middle-income countries. “We are pleased to support St. Jude in their efforts,” said Alain Baumann, CEO of the WFH. “Our hope is to contribute to a solution of limited clotting factor replacement therapy in resource-constrained countries.”
Glenn Pierce, MD, PhD, WFH Vice-President Medical added, “Presently, individuals in these countries have significant morbidity and mortality due to their hemophilia B. Gene therapy represents a new modality that can eliminate peaks and troughs of clotting factor replacement therapy. This results in circulating significant amounts of factor IX that should prevent most, or possibly all, bleeding episodes.”
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