ArticleJournal of inherited metabolic disease2006
Large neutral amino acids in the treatment of phenylketonuria (PKU).
Article in Journal of inherited metabolic disease, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 45 papers.
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Who cites it
45 citing papers in PubMed, 96 citations in OpenAlex.
- Double blind placebo control trial of large neutral amino acids in treatment of PKU: effect on blood phenylalanine.Journal of inherited metabolic disease · 2007Trial
- Foods for special medical purposes for the dietary therapy of rare diseases: Current status and future prospects.Intractable & rare diseases research · 2026Review
- A comprehensive integrated disease management program for phenylketonuria (IDMP-PKU) from Türkiye: rationale, design and patient characteristics.Orphanet journal of rare diseases · 2025Observational
- Current state of the treatment landscape of phenylketonuria.Orphanet journal of rare diseases · 2025Review
- Glycomacropeptide-Based Protein Substitutes for Children with Phenylketonuria in Italy: A Nutritional Comparison.Nutrients · 2024Review
- Glycomacropeptide in PKU-Does It Live Up to Its Potential?Nutrients · 2022Review
- Food Regime for Phenylketonuria: Presenting Complications and Possible Solutions.Journal of multidisciplinary healthcare · 2022Review
- The effect of casein glycomacropeptide versus free synthetic amino acids for early treatment of phenylketonuria in a mice model.PloS one · 2022Article
- Untreated PKU patients without intellectual disability:Molecular genetics and metabolism reports · 2021Article
- Beneficial Effects of Slow-Release Large Neutral Amino Acids after a Phenylalanine Oral Load in Patients with Phenylketonuria.Nutrients · 2021Article
- The Impact of a Slow-Release Large Neutral Amino Acids Supplement on Treatment Adherence in Adult Patients with Phenylketonuria.Nutrients · 2020Article
- Large Neutral Amino Acids (LNAAs) Supplementation Improves Neuropsychological Performances in Adult Patients with Phenylketonuria.Nutrients · 2020Article
- Large Neutral Amino Acid Therapy Increases Tyrosine Levels in Adult Patients with Phenylketonuria: A Long-Term Study.Nutrients · 2019Article
- Article
- Optimising amino acid absorption: essential to improve nitrogen balance and metabolic control in phenylketonuria.Nutrition research reviews · 2019Review
- Glycomacropeptide: long-term use and impact on blood phenylalanine, growth and nutritional status in children with PKU.Orphanet journal of rare diseases · 2019Review
- Psychiatric and Cognitive Aspects of Phenylketonuria: The Limitations of Diet and Promise of New Treatments.Frontiers in psychiatry · 2019Review
- An overview of inborn errors of metabolism affecting the brain: from neurodevelopment to neurodegenerative disorders.Dialogues in clinical neuroscience · 2018Review
- Cellular neurometabolism: a tentative to connect cell biology and metabolism in neurology.Journal of inherited metabolic disease · 2018Review
- Comparison of Glycomacropeptide with Phenylalanine Free-Synthetic Amino Acids in Test Meals to PKU Patients: No Significant Differences in Biomarkers, Including Plasma Phe Levels.Journal of nutrition and metabolism · 2018Article
Corrections and comments
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Authors and funding
9 authors at 6 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Large neutral amino acids (LNAAs) have been used on a limited number of patients with phenylketonuria (PKU) with the purpose of decreasing the influx of phenylalanine (Phe) to the brain. In earlier studies on mice with PKU (ENU(2)/ENU(2)), LNAAs were given and a surprising decline in blood Phe concentrations was observed. The formula used in the mouse experiment (PreKUnil) lacked lysine. Therefore, a new formulation of LNAAs (NeoPhe) was developed, introducing changes in the concentration of some amino acids and adding lysine, so that such a mixture could be used in humans. The new formula was found to be effective in reducing blood Phe concentration in mice by about 50% of the elevated levels. Patients with PKU were given LNAAs and blood Phe concentrations were determined in an open-label study. Three centers--in Russia, the Ukraine and the USA--took part in the study. NeoPhe was given at 0.5 g/kg per day in three divided doses to eight subjects with PKU and at 1.0 g/kg per day to three patients, for one week. The NeoPhe resulted in decrease of elevated blood Phe by 50% in both groups. The preliminary data from this study are encouraging and a double blind placebo-controlled trial will be required to show long-term efficacy and tolerance of LNAAs in the treatment of PKU.
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