BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) released topline data from the Phase 3 PEGASUS trial evaluating Palynziq ...

PKU is a rare, inherited metabolic disorder that prevents the body from breaking down the amino acid phenylalanine. Untreated phenylketonuria can lead to brain damage, intellectual disabilities ...

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 ...

The Argentina inborn errors of protein metabolism (IEPM) market was valued at US$ 268.2 million in 2023 and is expected to achieve a market valuation of US$ 664.0 million by 2032. This impressive ...

PKU is a genetic disease that manifests at birth and results from an inability to break down phenylalanine (phe), an amino acid that is commonly found in many foods. Left untreated, high levels of ...

Newborns with neonatal onset OTC deficiency experience symptoms of hyperammonemia shortly after birth, including lethargy, poor suck and vomiting, that if left untreated can quickly escalate to ...

“Untreated, these disorders can lead to multiorgan ... All 50 states screen newborns for phenylketonuria. Most states also test newborns for galactosemia. But no state tests babies for all ...

Among the most vital tests is screening for phenylketonuria (PKU), a rare metabolic disorder ... among the conditions that can be identified through early testing. “Severe untreated jaundice, for ...

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PALYNZIQ is the first and only enzyme therapy approved to treat adults with PKU. BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.) Detailed results from the PEGASUS study will ...

Sepiapterin has a dual mechanism of action to increase activity of the phenylalanine hydroxylase enzyme in patients with PKU, a rare, inherited metabolic disease. The new data revealed that over ...

Market OverviewThe global Newborn Screening Market is valued at USD 3.44 Billion in 2024 and is projected to reach a value of ...