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A gene therapy that could provide a permanent cure for sickle cell disease continues to show success through a third wave of patients, researchers report.
The therapy, LentiGlobin, restored normal blood function in 35 sickle cell patients who had the one-time procedure, according to clinical trial findings published Sunday in the New England Journal of Medicine.
The patients are all now producing stable amounts of normal red blood cells containing healthy hemoglobin, said lead researcher Dr. Julie Kanter, director of the University of Alabama at Birmingham’s Adult Sickle Cell Clinic.
In addition, they have not suffered the severe pain episodes that come with sickle cell disease, she said.
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news & eventsGlobal Blood Therapeutics Receives EMA PRIME Designation for GBT440 for the Treatment of Sickle Cell Disease (SCD)GBT440 is First Potential Treatment for ...
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.