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FDA advisers urge gene therapy trials despite bubble boy case
FDA advisers say gene therapy for SCID should resume with safeguards, noting a French toddler developed leukemia-like symptoms after successful treatment. They stress informed consent and long term monitoring, while confirming that more patients may access retroviral gene therapy if eligible.
MENTIONED IN THIS ARTICLE
Gaithersburg, Md
U.S
Philip Noguchi
Food and Drug Administration
FDA
Abby Meyers
National Organization For Rare Disorders
Congress
Crystal Mackall
National Cancer Institute
Joanne Kurtzberg
Duke University
David
Houston
Fischer
Christof Kalle
University of Cincinnati