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How phenylketonuria, a once-neglected disease, became a proving ground for new drugs

After decades of yearning for drugs, people with PKU now have 2, and a dozen more are in development

by Ryan Cross
July 8, 2019 | A version of this story appeared in Volume 97, Issue 27

Credit: Erik Jacobs | The Parrazzo and Ward families, who formed a friendship because they each have kids with PKU, cook a lowphenylalanine dinner at the Parrazzos’ house in Raynham, Massachusetts. Vinny Parrazzo (right) drinks his medical formula before dinner.

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