The company is advancing a robust pipeline of ENB-based therapeutics targeting both rare and prevalent chronic diseases, ...

The FDA has approved BioMarin’s pegvaliase-pqpz (Palynziq), a drug for the treatment of phenylketonuria (PKU) in adults. PKU is a rare autosomal recessive disorder, affecting approximately 1 in every ...

(MENAFN- GlobeNewsWire - Nasdaq) Phenylketonuria, which is characterized by the absence or deficiency of an enzyme called phenylalanine hydroxylase (PAH), responsible for processing the amino acid ...

Major companies such as Synlogic, Homology Medicines, PTC therapeutics, Rubius Therapeutics, NestlÃ, BioMarin, SOM Biotech, Evox Therapeutics, Moderna Therapeutics ...

Phenylketonuria is an inherited disease characterized by progressive mental retardation and seizures because the individual is deficient in the protein PAH. Most of the genetic mutations that cause ...

NORWICH, Conn. (WFSB) - Two siblings in Norwich have the same rare condition that severely limits their diet. 17-year-old Marguerite Brown has no choice but to avoid meat, too much could make her ...

Researchers at The University of Texas MD Anderson Cancer Center have discovered a critical new factor in regulating phenylalanine metabolism and, therefore, in preventing the inherited metabolic ...

Hyperphenylalaninemia is a common inherited metabolic disease that is due to phenylalanine hydroxylase deficiency, and at least half the affected patients have mild clinical phenotypes. Treatment with ...

PREVIOUS reports have indicated that a phenylalanine-deficient diet initiated in the early months of life may be effective in preventing the severe mental retardation commonly seen in patients with ...

We established correlations between APV and %PAH, FoldX, SNPs3D, PolyPhen-2 and SIFT Blink values. In a second step, multiple regression analysis was run to predict APV from the aforementioned values.

Phenylketonuria is the most common metabolic disease in humans. It occurs when the so-called PAH gene is defective, thus preventing the amino acid phenylalanine from being metabolized to the amino ...

New York, USA, April 29, 2025 (GLOBE NEWSWIRE) -- Phenylketonuria Clinical Trial Pipeline Appears Robust With 25+ Key Pharma Companies Actively Working in the Therapeutics Segment | DelveInsight | ...