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Doctors Treat Girl s Genetic Disorder with Repurposed Drug
In just 16 months, physicians went from identifying a novel rare disease in three-year-old Marley to successfully treating her with a drug previously used to treat African sleeping sickness and pediatric cancer.
Michigan state university
United states
Kwame anyane yeboa
Anne pariser
Johns hopkins
Tracy murray stewart
Kelly berthoud
Caleb bupp
Surender rajasekaran
National institutes of health
Office of rare diseases research
Columbia university medical center
Spectrum health
Helen devos children
Johns hopkins medicine
Rare diseases research
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