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Education and empowerment would provide more equal access to care

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Ella, 28, was diagnosed with cystic fibrosis – a rare genetic disease which causes a build-up of thick, sticky mucus in the lungs, digestive tract, and sinuses – at 18 months old. This interrupts replication patterns and, ultimately, kills the bacterium.”. The antibiotics I was taking intravenously were not helping at all.

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Synlogic preps phenylketonuria drug for phase 3 after mid-stage win

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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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