BEDFORD, Mass., July 27, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (FIXX), a genetic medicines company, announced today encouraging initial clinical data from the first dose cohort in the ...
Phenylketonuria (PKU) is a rare genetic condition that affects how your body processes an amino acid called phenylalanine (Phe). People with PKU have low levels or missing activity of an enzyme called ...
What Is Sephience, and Why Does It Matter? Sephience (sepiapterin) is a new oral medication approved by the FDA to treat a rare inherited condition known as phenylketonuria (PKU) in adults and ...
To control their phenylalanine (Phe) levels, patients with PKU are advised to follow a restricted diet. However, little is known about the long-term consequences. A new study of 15 adults with PKU ...
People with phenylketonuria cannot break down a substance in food called phenylalanine. Following a phenylketonuria diet is the main treatment for preventing serious symptoms. People with ...
Newborn screening for PKU has allowed for early management of the condition, which serves to improve the prognosis in most cases. Research into PKU in adults has focused mostly on cognitive function; ...
Sassy Sister Vibes on MSN
Woman adopts child with the same rare metabolic disorder
Growing up with a rare genetic disorder, Pam Kowalczyk often felt like no one truly understood her daily struggles. Years ...
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