Tag: Sickle Cell Disease

New sickle cell treatment has roots at Children’s Hospital of Philadelphia
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New sickle cell treatment has roots at Children’s Hospital of Philadelphia

Sickle cell therapy pioneered at CHOP a "real gamechanger" Sickle cell therapy pioneered at CHOP a "real gamechanger" 02:40 PHILADELPHIA (CBS) – A new medical breakthrough has roots in Philadelphia. The Food and Drug Administration recently approved the first gene therapies to treat patients with sickle cell disease, an inherited blood disorder that strikes mainly people of color. "You feel like somebody is taking a sledgehammer to your bones and hitting it over and over again," said...
New sickle cell treatment has roots at Children’s Hospital of Philadelphia
Health

New sickle cell treatment has roots at Children’s Hospital of Philadelphia

PHILADELPHIA (CBS) – A new medical breakthrough has roots in Philadelphia. The Food and Drug Administration recently approved the first gene therapies to treat patients with sickle cell disease, an inherited blood disorder that strikes mainly people of color. "You feel like somebody is taking a sledgehammer to your bones and hitting it over and over again," said Daniels Tornyenu, who has sickle cell disease. His daughter, Marie-Chantal Tornyenu, has the illness as well. "In my chest, it's like this hollow burning almost, and then I can't breathe on top of it," Marie-Chantal Tornyenu said. RELATED: FDA approves gene-editing treatment for sickle cell diseasePain from sickle cell can happen anywhere blood circulates. Because red blood cells, which are normally round, bend into inflexible s...
FDA approves first gene therapy for sickle cell disease
Health

FDA approves first gene therapy for sickle cell disease

FDA approves first gene therapy for sickle cell disease - CBS News Watch CBS News The Food and Drug Administration on Friday approved the first-ever gene-editing therapy that uses a cutting-edge technology called CRISPR to treat sickle cell disease. Dr. Jon LaPook has more. Be the first to know Get browser notifications for breaking news, live events, and exclusive reporting. Not Now Turn On
World’s first gene therapy for sickle cell and thalassemia approved in the U.K.
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World’s first gene therapy for sickle cell and thalassemia approved in the U.K.

Expert joins CBS New York to discuss new sickle cell treatment Expert joins CBS New York to discuss new sickle cell treatment 02:46 Britain's medicines regulator has authorized the world's first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the U.K.In a statement on Thursday, the Medicines and Healthcare Regulatory Agency said it approved Casgevy, the first medicine licensed using the gene editing ...