Treatment with PALYNZIQ led to statistically significant blood phenylalanine (Phe) lowering compared to diet alone in pivotal ...
The FDA expands Palynziq approval to include pediatric patients aged 12 years and older with phenylketonuria and uncontrolled blood Phe levels.
Growing up with a rare genetic disorder, Pam Kowalczyk often felt like no one truly understood her daily struggles. Years ...
The approval follows PEGASUS Phase III trial assessing Palynziq efficacy and safety compared to diet alone in adolescents ...
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 ...
Credit: Getty Images. The investigational therapy is an oral formulation of synthetic sepiapterin. Sepiapterin significantly reduced blood phenylalanine levels in adult and pediatric patients with ...
—Researchers in Germany recently analyzed several parameters related to bone metabolism in plasma, serum, and urine—as well as tracked dietary habits and lifestyle factors—in a cohort of adults with ...
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