Stem Cell Therapy for Sickle Cell
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revolutionary sickle cell gene therapy processNBC News
•'Will it really work?': Young sickle cell patient among the first to start new gene therapy
71% Informative
Wedam Minyila , 19 , is one of the first patients in the world to undergo treatment for sickle cell disease.
He's one of only a few dozen patients nationwide who have been able to access it.
The FDA approved two gene therapies that could cure the symptoms of the disease just over a year ago .
Only a limited number of hospitals have treated patients so far.
At Children’s National , only 10 people, including Wedam , have initiated or finished the process.
The monthslong treatment Casgevy from Vertex Pharmaceuticals comes with a list price of $2.3 million .
The intensive chemotherapy lasts four days and makes patients extremely ill and susceptible to infection.
Until the gene therapy, the only cure for sickle cell disease was a stem cell transplant from a donor.
After some scary complications, the procedure was successful, but antibodies in Wedam ’s bloodstream prevented the same cure for him.
“I feel like it changed the life that he is going to live,” Wekem Minyila said.
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