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.

The Food and Drug Administration (FDA) has granted Orphan Drug designation to PTC923 for the treatment of patients with hyperphenylalaninemia. Hyperphenylalaninemia, a rare metabolic condition ...

Dec. 26, 2002 -- Tetrahydrobiopterin may be a useful alternative to dietary restriction for mild hyperphenylalanemia or mild phenylketonuria (PKU), according to a preliminary report published in ...

Phenylketonuria (PKU) accounts for 98% of all hyperphenylalaninemia cases and is a metabolic condition that can lead to cognitive disabilities and seizures. PTC923 is an oral formulation of ...

CHMP Issued Positive Opinion Recommending Marketing Authorization of Kuvan(R) for the Treatment of hyperphenylalaninemia in Patients With phenylketonuria or BH4 Deficiency Merck Serono ...

First Drug to be Approved in Europe for the Treatment of Hyperphenylalaninemia due to Phenylketonuria or BH4 Deficiency Merck Serono, a division of Merck KGaA, Darmstadt, Germany, today announced ...

Scriver CR, Kaufman S . Hyperphenylalaninemia: phenylalanine hydroxylase deficiency. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds) The Metabolic & Molecular Bases of Inherited Disease.

have granted an Orphan Drug designation to its pipeline candidate PTC923 which is being developed for the treatment of patients with hyperphenylalaninemia. The company plans to begin a placebo ...

Characterization of mutant genes associated with hereditary diseases is aimed primarily at understanding the molecular basis of these disorders. However, identification of sequence variations ...

GlobalData's clinical trial report, "Hyperphenylalaninemia Global Clinical Trials Review, H2, 2017" provides an overview of Hyperphenylalaninemia clinical trials scenario. This report provides top ...

The latest research report Hyperphenylalaninemia (HPA) Market, covers various aspects of the industry, including market size, status, trends, and future insights for the next 10 years. It provides a ...