ReviewMetabolites2014
New Strategies for the Treatment of Phenylketonuria (PKU).
Review in Metabolites, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
16 citing papers in PubMed.
- Protein substitutions as new-generation pharmanutrition approach to managing phenylketonuria.Clinical and experimental pediatrics · 2023Article
- Review
- Protein Degradation and the Pathologic Basis of Phenylketonuria and Hereditary Tyrosinemia.International journal of molecular sciences · 2020Review
- Strategies to engage lost to follow-up patients with phenylketonuria in the United States: Best practice recommendations.Molecular genetics and metabolism reports · 2020Article
- Genome editing technologies to treat rare liver diseases.Translational gastroenterology and hepatology · 2020Review
- Large neutral amino acid status in association with P:T ratio and diet in adult and pediatric patients with phenylketonuria.JIMD reports · 2019Article
- Psychiatric and Cognitive Aspects of Phenylketonuria: The Limitations of Diet and Promise of New Treatments.Frontiers in psychiatry · 2019Review
- Import of TAT-Conjugated Propionyl Coenzyme A Carboxylase Using Models of Propionic Acidemia.Molecular and cellular biology · 2018Article
- Effect of natural PAL-enzyme on the quality of egg white and mushroom flour and study its impact on the expression of PKU related genes and phenylalanine reduction in mice fed on.Journal, genetic engineering & biotechnology · 2017Article
- CRISPR RNA-guided FokI nucleases repair a PAH variant in a phenylketonuria model.Scientific reports · 2016Article
- Molecular epidemiology, genotype-phenotype correlation and BH4 responsiveness in Spanish patients with phenylketonuria.Journal of human genetics · 2016Article
- Protein substitutes for phenylketonuria in Europe: access and nutritional composition.European journal of clinical nutrition · 2016Article
- Dietary intervention in the management of phenylketonuria: current perspectives.Pediatric health, medicine and therapeutics · 2016Review
- Phenylketonuria and Gut Microbiota: A Controlled Study Based on Next-Generation Sequencing.PloS one · 2016Article
- Intellectual and Developmental Status in Children With Hyperphenylalaninemia and PKU Who Were Screened in a National Program.Iranian journal of pediatrics · 2015Article
- Emerging trends in nutraceutical applications of whey protein and its derivatives.Journal of food science and technology · 2015Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Phenylketonuria (PKU) was the first inherited metabolic disease in which dietary treatment was found to prevent the disease's clinical features. Treatment of phenylketonuria remains difficult due to progressive decrease in adherence to diet and the presence of neurocognitive defects despite therapy. This review aims to summarize the current literature on new treatment strategies. Additions to treatment include new, more palatable foods based on glycomacropeptide that contains very limited amount of aromatic amino acids, the administration of large neutral amino acids to prevent phenylalanine entry into the brain or tetrahydropterina cofactor capable of increasing residual activity of phenylalanine hydroxylase. Moreover, human trials have recently been performed with subcutaneous administration of phenylalanine ammonia-lyase, and further efforts are underway to develop an oral therapy containing phenylanine ammonia-lyase. Gene therapy also seems to be a promising approach in the near future.
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.