8-year-old from Valley Stream becomes first in NY to undergo gene therapy for rare disease
It involved removing stem cells from Yusef, genetically altering them and then putting them back into his body with the hope they would cure the disease.
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An 8-year-old from Valley Stream has become the first person in New York to undergo gene therapy for a rare disease.
Yusef Ahmed was diagnosed with thalassemia – a rare, genetic blood disease – when he was 1 month old.
He has had to undergo red blood cell transfusion every few weeks to avoid long-term illness or worse.
In January, his parents decided to try gene therapy treatment, which was administered for the first time in New York state at Cohen Children’s Medical Center in New Hyde Park.
It involved removing stem cells from Yusef, genetically altering them and then putting them back into his body with the hope they would cure the disease.
On Thursday, doctors spoke about the amazing progress Yusef has made, and how he has not needed a transfusion since February.
They are hopeful he may not need one again.
