Search GLEAM-DB / CoGenEx-PM3

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Input query
Recognized gene
PAH
Normalized c.HGVS
c.1003A>G, c.1010G>T, c.1030G>C, c.1031G>T, c.1066-3C>G and 44 more
Normalized p.HGVS
p.(=), p.(Ala165Pro), p.(Ala246Asp), p.(Arg157Thr), p.(Arg158Pro) and 38 more
Matching records
414
PM3-positive records
69

Results are ordered by PM3-relevant evidence first. Reviewed source only indicates whether main text, supplementary material, or both were reviewed; it is not the evidence conclusion.

Download current query results as TSV
Gene Variant Evidence PM3 context Publication Reviewed source Actions
PAH NM_000277.3:c.158G>A Phase-confirmed PM3 evidence
High confidence
Confirmed in trans with c.728G > A
context: Confirmed in trans
36333673
Mild hyperphenylalaninemia (hpa) presenting as orthostatic tremor: a case report.
BMC neurology, 2022
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.728G>A; p.R243Q
context: Compound heterozygous candidate
41413260
Newborn screening for inherited metabolic disorders in central China: a retrospective study of 153,956 infants using non-derivatized tandem mass spectrometry.
Scientific reports, 2025
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.721C>T; c.728G>A; p.Arg241Cys; +1 more
context: Compound heterozygous candidate
41357791
Incidence and disease spectrum of inherited metabolic diseases screened by tandem mass spectrometry in Huai'an from 2018 to 2024.
Frontiers in pediatrics, 2025
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Confirmed in trans with c.60+4A>G; p.?; splicing
context: Confirmed in trans
41291872
The incidence rate and gene mutation characteristics of hyperphenylalaninemia in Yunnan Province, Southwest China.
Orphanet journal of rare diseases, 2025
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.631C>A; p.Pro211Thr
context: Compound heterozygous candidate
40293582
Genotypic and phenotypic characteristics of Turkish patients with phenylalanine metabolism disorders.
Metabolic brain disease, 2025
Main article
Open
PAH NM_000277.3:c.168+5G>T Phase-unconfirmed biallelic evidence
Needs review
Homozygous for query variant
context: Homozygous evidence
39940237
Genotype-Phenotype Correlation in a Large Cohort of Eastern Sicilian Patients Affected by Phenylketonuria: Newborn Screening Program, Clinical Features, and Follow-Up.
Nutrients, 2025
Main article
Open
PAH NM_000277.3:c.781C>G Phase-unconfirmed biallelic evidence
Needs review
Homozygous for query variant
context: Homozygous evidence
39940237
Genotype-Phenotype Correlation in a Large Cohort of Eastern Sicilian Patients Affected by Phenylketonuria: Newborn Screening Program, Clinical Features, and Follow-Up.
Nutrients, 2025
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.688G>A
context: Compound heterozygous candidate
39969324
Exploring a novel model for newborn genetic screening in Ningxia, northern China: A retrospective observational study.
Medicine, 2024
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.547G>T; c.611A>G; c.728G>A; +5 more
context: Compound heterozygous candidate
39776477
Application of targeted high-throughput sequencing as a diagnostic tool for neonatal genetic metabolic diseases following tandem mass spectrometry screening.
Frontiers in public health, 2024
Supplementary material
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.1238G>C; c.526C>T; c.688G>A; +13 more
context: Compound heterozygous candidate
39670100
Spectrum analysis of inborn errors of metabolism for expanded newborn screening in Xinjiang, China.
PeerJ, 2024
Supplementary material
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.1315+6T>A
context: Compound heterozygous candidate
38577637
The significance of machine learning in neonatal screening for inherited metabolic diseases.
Frontiers in pediatrics, 2024
Main article
Open
PAH NM_000277.3:c.689T>G Phase-unconfirmed biallelic evidence
Needs review
Confirmed in trans with c.688G> A; p.V230I
context: Confirmed in trans
38481932
Identified PAH V230A and PAH V230I mutations in a family with diverse clinical presentations.
Clinical case reports, 2024
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Potential PM3 evidence identified; genotype context not structured
context: Compound heterozygous candidate
38105703
Analysis of gene variation and long-term follow-up in children with phenylalanine hydroxylase deficiency diagnosed by newborn screening.
Zhejiang da xue xue bao. Yi xue ban = Journal of Zhejiang University. Medical sciences, 2023
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.1199+502A>T
context: Compound heterozygous candidate
37237386
Identification of deep intronic variants of PAH in phenylketonuria using full-length gene sequencing.
Orphanet journal of rare diseases, 2023
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Homozygous for query variant
context: Homozygous evidence
37098607
The spectrum of phenylalanine hydroxylase variants and genotype-phenotype correlation in phenylketonuria patients in Gansu, China.
Human genomics, 2023
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Potential PM3 evidence identified; genotype context not structured
context: Compound heterozygous candidate
37004080
Allelic phenotype prediction of phenylketonuria based on the machine learning method.
Human genomics, 2023
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Homozygous for query variant
context: Homozygous evidence
36927542
A retrospective analysis of MS/MS screening for IEM in high-risk areas.
BMC medical genomics, 2023
Main article
Open
PAH NM_000277.3:c.473G>C Phase-unconfirmed biallelic evidence
Needs review
Possible compound heterozygous with c.1243G>A; c.827T>C
context: Compound heterozygous candidate
36768810
Comparison of Tandem Mass Spectrometry and the Fluorometric Method-Parallel Phenylalanine Measurement on a Large Fresh Sample Series and Implications for Newborn Screening for Phenylketonuria.
International journal of molecular sciences, 2023
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Homozygous for query variant
context: Homozygous evidence
36246604
Expanded newborn screening for inherited metabolic disorders by tandem mass spectrometry in a northern Chinese population.
Frontiers in genetics, 2022
Main article
Open
PAH NM_000277.3:c.158G>A Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.1073 T > G; c.1138del; c.1162G > A; +21 more
context: Compound heterozygous candidate
36104584
Characterization of phenylalanine hydroxylase gene variants and analysis of genotype-phenotype correlation in patients with phenylalanine hydroxylase deficiency from Fujian Province, Southeastern China.
Molecular biology reports, 2022
Main article
Open