New Sickle Cell Treatment Has Roots At Children's Hospital Of Philadelphia

By Stephanie Stahl, CBS Philadelphia

December 12, 2023

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.

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