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Gene Therapy Clinical Study in Adult PKU

2019-05-19 19:37:15 | BioPortfolio

Published on BioPortfolio: 2019-05-19T19:37:15-0400

Clinical Trials [9 Associated Clinical Trials listed on BioPortfolio]

Evaluation of PKU Sphere

A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet.

A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.

Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase

Conduct a prospective, longitudinal study to evaluate nutritional status in adults with phenylketonuria (PKU) before and during treatment with pegvaliase (Palynziq™).

A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU

This study aims to measure changes in participants' blood phenylalanine (Phe) levels in participants with PKU when switching from a conventional amino acid based protein substitute to a GM...

Nutrition Status of Adults Treated With Pegvaliase

Conduct a cross-sectional analysis of the nutritional status of individuals with PKU treated with pegvaliase for greater than one year. We will evaluate laboratory markers of nutritional s...

PubMed Articles [0 Results]

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Medical and Biotech [MESH] Definitions

An enzyme of the oxidoreductase class that catalyzes the formation of L-TYROSINE, dihydrobiopterin, and water from L-PHENYLALANINE, tetrahydrobiopterin, and oxygen. Deficiency of this enzyme may cause PHENYLKETONURIAS and PHENYLKETONURIA, MATERNAL. EC 1.14.16.1.

A group of autosomal recessive disorders marked by a deficiency of the hepatic enzyme PHENYLALANINE HYDROXYLASE or less frequently by reduced activity of DIHYDROPTERIDINE REDUCTASE (i.e., atypical phenylketonuria). Classical phenylketonuria is caused by a severe deficiency of phenylalanine hydroxylase and presents in infancy with developmental delay; SEIZURES; skin HYPOPIGMENTATION; ECZEMA; and demyelination in the central nervous system. (From Adams et al., Principles of Neurology, 6th ed, p952).

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