Doctors make history using novel technique to treat fatal genetic disease before birth

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Doctors make history using novel technique to treat fatal genetic disease before birth
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Doctors make history using novel technique to treat fatal genetic disease before birth FOX13

Wyatt Shield talks with FOX Television Stations about being a published author at 6 years old.

"She’s just a regular little 1½-year-old who keeps us on our toes," Bashir said. The couple previously lost two daughters, Zara, 2½, and Sara, 8 months, to the disease. A third pregnancy was terminated because of the disorder.in the New England Journal of Medicine, doctors describe an international collaboration during the COVID-19 pandemic that led to the treatment that may have saved Ayla’s life – and expanded the field of potential fetal therapies.

The unusual partnership also involved experts at Duke University in Durham, N.C., which has led research on Pompe disease, and University of Washington in Seattle. In late 2020, Bashir and Qureshi had learned they were expecting Ayla and that prenatal tests showed she, too, had Pompe disease. Chakraborty had learned of MacKenzie’s early stage trial to test the enzyme therapy and thought early treatment might be a solution for the family.

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