Synlogic preps phenylketonuria drug for phase 3 after mid-stage win
pharmaphorum
OCTOBER 19, 2022
PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down phenylalanine (phe), an amino acid that is commonly found in many foods. Left untreated, high levels of the amino acid become toxic to the brain and may lead to serious neurological and neuropsychological issues.
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