World’s First Gene Therapy for Glycogen Storage Disease Produces Remarkable Results - UConn Today
uconn.edu
World’s First Gene Therapy for Glycogen Storage Disease Produces Remarkable Results - UConn Today
The clinical trial originally set out to simply test the safety and dosage of the gene therapy for three patients with GSD Type Ia. The dramatic improvement ...
Sep 19, 2019
Gene Therapy Success Stories
Watch a new Double Take video: “Sickle Cell Disease and Gene Therapy.” In this short documentary, patients share their own stories of life with sickle cell disease and physicians disclose a cautious optimism about the benefits and risks of new treatment strategies, including gene therapy. Watch at https://youtu.be/A4ZMmPx7ZK8. | The New England Journal of Medicine
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Watch a new Double Take video: “Sickle Cell Disease and Gene Therapy.” In this short documentary, patients share their own stories of life with sickle cell disease and physicians disclose a cautious optimism about the benefits and risks of new treatment strategies, including gene therapy. Watch at https://youtu.be/A4ZMmPx7ZK8. | The New England Journal of Medicine
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