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Leslie Raffini, MD, and Regina Butler, RN, were quoted in the Philadelphia Inquirer on a story featuring a CHOP patient family living with hemophilia, an inherited bleeding disorder that prevents blood from clotting properly.
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A Penn Medicine and CHOP team will seek to develop treatments for three rare, incurable genetic diseases with the help of a $26 million grant from the National Institutes of Health.
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CHOP gene therapy expert Dr. Lindsey George reacts to a phase 3 hemophilia gene therapy study in an editorial in the New England Journal of Medicine.
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CHOP will be a provider in Independence Blue Cross’s new Advanced Network for Gene-Based Therapeutics, effective March 1, 2023.
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Two new treatments are giving children with spinal muscular atrophy hope. CHOP is on the forefront of delivering care that is changing children’s lives.
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Find out how a bold idea led to an astonishing cure for a certain childhood blindness, and learn about the future of gene therapy to treat children, like Hannah.
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Dr. George was recognized for the first gene therapy clinical trial to report a cure of hemophilia B patients.
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Diagnosed with Leber congenital amaurosis as a baby, Hannah was on a path to going blind until gene therapy at Children’s Hospital restored her sight.
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A breakthrough therapy has its genesis in more than 10 years of research conducted at Children's Hospital of Philadelphia.