PITTSBURGH (Newsradio 1020 KDKA) – Researchers at UPMC Children’s Hospital of Pittsburgh have successfully treated rare genetic disorders with umbilical cord blood.
The study, the largest trial of its kind to date, treated 20 different rare congenital diseases by infusing banked umbilical cord blood.
In the study, 44 children born with various non-cancerous genetic disorders, including sickle cell, thalassemia, Hunter syndrome, Krabbe disease, metachromatic leukodystrophy (MLD) and an array of immune deficiencies, were safely and effectively treated.
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Instead of chasing individual therapies for each of the rare diseases, researchers created a fairly universal treatment.
“There has been a lot of emphasis placed on cool new technologies that might address these diseases, but—even if they prove effective—those aren’t available to most centers,” study senior author Paul Szabolcs, M.D. said. “The regimen we developed is more robust, readily applicable and will remain significantly less expensive.”
To learn more about the study, click here.
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