Feingold syndrome type 1

disease
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Also known as Brunner-Winter syndrome type 1digital anomalies with short palpebral fissures and atresia of esophagus or duodenum type 1digital anomalies with short palpebral fissures and atresia of oesophagus or duodenumdigital anomalies with short palpebral fissures and atresia of oesophagus or duodenum type 1Feingold syndrome 1Feingold syndrome caused by mutation in MYCNFGLDS1FS1microcephaly, mental retardation, and tracheoesophageal fistula syndromemicrocephaly-digital anomalies-normal intelligence syndrome type 1microcephaly-intellectual disability-tracheoesophageal fistula syndrome type 1microcephaly-oculo-digito-esophageal-duodenal syndrome syndrome type 1MMT type 1MODED syndrome type 1MYCN Feingold syndromeoculo-digito-esophageal-duodenal syndrome type 1ODED syndrome type 1

Summary

Feingold syndrome type 1 (MONDO:0008115) is a disease caused by MYCN (GenCC Definitive), with 3 cohort genes.

At a glance

  • Prevalence: <1 / 1 000 000 (Worldwide) [Orphanet-validated]
  • Causal gene: MYCN (GenCC Definitive)
  • Cohort genes: 3
  • ClinVar variants: 123
  • Phenotypes (HPO): 35

Clinical features

Epidemiology

Prevalence records

2 prevalence record(s), Orphanet:

TypeClassValueGeographyValidation
Cases/families120WorldwideValidated
Point prevalence<1 / 1 000 000WorldwideValidated

Signs & symptoms

Clinical features (HPO)

35 HPO clinical features (Orphanet curated; top 35 by frequency):

HPO IDTermFrequency
HP:0000252MicrocephalyVery frequent (80-99%)
HP:0001770Toe syndactylyVery frequent (80-99%)
HP:0002589Gastrointestinal atresiaVery frequent (80-99%)
HP:0005819Short middle phalanx of fingerVery frequent (80-99%)
HP:0000347MicrognathiaFrequent (30-79%)
HP:0001328Specific learning disabilityFrequent (30-79%)
HP:0001999Abnormal facial shapeFrequent (30-79%)
HP:0002032Esophageal atresiaFrequent (30-79%)
HP:0004209Clinodactyly of the 5th fingerFrequent (30-79%)
HP:0004220Short middle phalanx of the 5th fingerFrequent (30-79%)
HP:00046912-3 toe syndactylyFrequent (30-79%)
HP:00046924-5 toe syndactylyFrequent (30-79%)
HP:0009577Short middle phalanx of the 2nd fingerFrequent (30-79%)
HP:0009778Short thumbFrequent (30-79%)
HP:0012745Short palpebral fissureFrequent (30-79%)
HP:0000076Vesicoureteral refluxOccasional (5-29%)
HP:0000077Abnormality of the kidneyOccasional (5-29%)
HP:0000083Renal insufficiencyOccasional (5-29%)
HP:0000085Horseshoe kidneyOccasional (5-29%)
HP:0000110Renal dysplasiaOccasional (5-29%)
HP:0000123NephritisOccasional (5-29%)
HP:0000126HydronephrosisOccasional (5-29%)
HP:0000405Conductive hearing impairmentOccasional (5-29%)
HP:0000407Sensorineural hearing impairmentOccasional (5-29%)
HP:0001627Abnormal heart morphologyOccasional (5-29%)
HP:0001643Patent ductus arteriosusOccasional (5-29%)
HP:0002247Duodenal atresiaOccasional (5-29%)
HP:0010446Tricuspid stenosisOccasional (5-29%)
HP:0011611Interrupted aortic archOccasional (5-29%)
HP:0011625Multiple muscular ventricular septal defectsOccasional (5-29%)
HP:0011662Tricuspid atresiaOccasional (5-29%)
HP:0001249Intellectual disabilityVery rare (<1-4%)
HP:0002023Anal atresiaVery rare (<1-4%)
HP:0004322Short statureVery rare (<1-4%)
HP:0005235Jejunal atresiaVery rare (<1-4%)

Identifiers

Disease identifiers

FieldValue
Canonical nameFeingold syndrome type 1
Mondo IDMONDO:0008115
OMIM164280
Orphanet391641
SNOMED CT702431004
UMLSC4551774
MedGen1637716
GARD0017624
Is cancer (heuristic)no

Also known as: Brunner-Winter syndrome type 1 · digital anomalies with short palpebral fissures and atresia of esophagus or duodenum type 1 · digital anomalies with short palpebral fissures and atresia of oesophagus or duodenum · digital anomalies with short palpebral fissures and atresia of oesophagus or duodenum type 1 · Feingold syndrome 1 · Feingold syndrome caused by mutation in MYCN · Feingold syndrome type 1 · FGLDS1 · FS1 · microcephaly, mental retardation, and tracheoesophageal fistula syndrome · microcephaly-digital anomalies-normal intelligence syndrome type 1 · microcephaly-intellectual disability-tracheoesophageal fistula syndrome type 1 · microcephaly-oculo-digito-esophageal-duodenal syndrome syndrome type 1 · MMT type 1 · MODED syndrome type 1 · MYCN Feingold syndrome · oculo-digito-esophageal-duodenal syndrome type 1 · ODED syndrome type 1

Data availability: 123 ClinVar variants · 5 GenCC gene-disease records.

Disease family

Classification path: disease › human disease › disease by etiologic mechanism › disease of genetic or genomic mechanism › hereditary disease › autosomal genetic disease › autosomal dominant disease › Feingold syndromeFeingold syndrome type 1

Related subtypes (1): Feingold syndrome type 2

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

123 retrieved; paginated sample, class counts are floors:

64 uncertain significance, 18 pathogenic, 14 likely pathogenic, 11 conflicting classifications of pathogenicity, 8 likely benign, 5 pathogenic/likely pathogenic, 2 not provided, 1 benign/likely benign

ClinVarVariant (HGVS)GeneClassificationReview
1323303NM_005378.6(MYCN):c.559del (p.Val187fs)MYCNPathogeniccriteria provided, single submitter
13892NM_005378.6(MYCN):c.1178G>A (p.Arg393His)MYCNPathogeniccriteria provided, multiple submitters, no conflicts
13893NM_005378.6(MYCN):c.1177C>A (p.Arg393Ser)MYCNPathogenicno assertion criteria provided
13894NM_005378.6(MYCN):c.1181G>A (p.Arg394His)MYCNPathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
13896NM_005378.6(MYCN):c.217G>T (p.Glu73Ter)MYCNPathogenicno assertion criteria provided
13897NM_005378.6(MYCN):c.626dup (p.Ala210fs)MYCNPathogeniccriteria provided, single submitter
1709269NM_005378.6(MYCN):c.256G>T (p.Glu86Ter)MYCNPathogeniccriteria provided, single submitter
1710469NM_005378.6(MYCN):c.403C>T (p.Gln135Ter)MYCNPathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
2108584NM_005378.6(MYCN):c.621dup (p.Ala208fs)MYCNPathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
2507183NM_005378.6(MYCN):c.134del (p.Pro45fs)MYCNPathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
3376293NM_005378.6(MYCN):c.833_846del (p.Asp278fs)MYCNPathogeniccriteria provided, single submitter
433149NM_005378.6(MYCN):c.1014C>A (p.Tyr338Ter)MYCNPathogeniccriteria provided, single submitter
433150NM_005378.6(MYCN):c.68_71dup (p.Gln25fs)MYCNPathogeniccriteria provided, single submitter
433151NM_005378.6(MYCN):c.964C>T (p.Arg322Ter)MYCNPathogeniccriteria provided, multiple submitters, no conflicts
433152NM_005378.6(MYCN):c.1061dup (p.Ser355fs)MYCNPathogeniccriteria provided, single submitter
433154NM_005378.6(MYCN):c.902_903del (p.Val301fs)MYCNPathogeniccriteria provided, single submitter
4526864NM_005378.6(MYCN):c.985C>T (p.Gln329Ter)MYCNPathogeniccriteria provided, single submitter
4796616NM_005378.6(MYCN):c.411_417delinsTTCCA (p.Arg138fs)MYCNPathogeniccriteria provided, single submitter
545970NM_005378.6(MYCN):c.1117C>T (p.Arg373Ter)MYCNPathogeniccriteria provided, multiple submitters, no conflicts
817596NM_005378.6(MYCN):c.134dup (p.Glu47fs)MYCNPathogeniccriteria provided, multiple submitters, no conflicts
13895NM_005378.6(MYCN):c.231G>A (p.Trp77Ter)MYCNOSPathogenicno assertion criteria provided
2571637NM_005378.6(MYCN):c.367del (p.Arg123fs)MYCNOSPathogeniccriteria provided, single submitter
180573NM_001267550.2(TTN):c.67495C>T (p.Arg22499Ter)TTN-AS1Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
1185065NM_005378.6(MYCN):c.796G>T (p.Glu266Ter)MYCNLikely pathogenicno assertion criteria provided
1285553NM_005378.6(MYCN):c.152del (p.Lys51fs)MYCNLikely pathogeniccriteria provided, single submitter
1679249NM_005378.6(MYCN):c.77C>T (p.Pro26Leu)MYCNLikely pathogeniccriteria provided, single submitter
2499579NM_005378.6(MYCN):c.1274_1283del (p.Lys425fs)MYCNLikely pathogeniccriteria provided, single submitter
2506946NM_005378.6(MYCN):c.1211T>G (p.Leu404Arg)MYCNLikely pathogeniccriteria provided, single submitter
2581158NM_005378.6(MYCN):c.633_634dup (p.Ala212fs)MYCNLikely pathogeniccriteria provided, single submitter
2627009NM_005378.6(MYCN):c.727C>T (p.Gln243Ter)MYCNLikely pathogeniccriteria provided, single submitter

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 6 · Orphanet: 4 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

GenCC gene–disease validity (cohort genes)

the Disease column is the GenCC-asserted condition — a cohort gene’s strongest validity may be for a related predisposition syndrome.

GeneClassificationInheritanceDiseaseRecords
MYCNDefinitiveAutosomal dominantFeingold syndrome type 16

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
MYCNOrphanet:357027Hereditary retinoblastoma
MYCNOrphanet:357034Non-hereditary retinoblastoma
MYCNOrphanet:391641Feingold syndrome type 1
MYCNOrphanet:635Neuroblastoma

Cohort genes → proteins

3 cohort genes, 2 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence3

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
MYCNHGNC:7559ENSG00000134323P04198N-myc proto-oncogene proteingencc,clinvar
MYCNOSHGNC:16911ENSG00000233718P40205N-cym proteinclinvar
TTN-AS1HGNC:44124ENSG00000237298TTN antisense RNA 1clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
MYCNN-myc proto-oncogene proteinPositively regulates the transcription of MYCNOS in neuroblastoma cells.
MYCNOSN-cym proteinRegulates stability of MYCN in neuroblastoma cells by inhibiting GSK3B-mediated MYCN phosphorylation.

Protein-family classification

Druggable: 0 · Difficult: 1 · Unknown: 2 · Druggable fraction: 0.0

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

FamilyGenesFoldFDR
Transcription factor12.8×0.587
Other/Unknown21.2×0.587

Per-gene assignment

SymbolFamilyDruggable?ECInterPro (top 3)
MYCNTranscription factornoTscrpt_reg_Myc, bHLH_dom, Tscrpt_reg_Myc_N
MYCNOSOther/Unknownno
TTN-AS1Other/Unknownno

Expression context

Cohort genes with no expression data: 0.

2 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

BucketGenes
narrow (1-5 tissues)0
moderate (6-20)0
broad (>20)3
unknown0

Top tissues across cohort

TissueCohort genes
cortical plate2
ventricular zone2
embryo1
primordial germ cell in gonad1
gastrocnemius1
hindlimb stylopod muscle1
right atrium auricular region1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
MYCN223broadyesventricular zone, cortical plate, embryo
MYCNOS153tissue_specificmarkerprimordial germ cell in gonad, cortical plate, ventricular zone
TTN-AS1174ubiquitousmarkerhindlimb stylopod muscle, gastrocnemius, right atrium auricular region

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
MYCN7,345
MYCNOS0
TTN-AS10

Structural data

PDB: 1 · AlphaFold-only: 1 · No structure: 1

Cohort genes with PDB structures (top 30)

SymbolUniProtPDB entries
MYCNP041982

AlphaFold-only cohort genes (top 30 by pLDDT)

SymbolUniProtpLDDT
MYCNOSP4020548.22

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 8. Enrichment computed across 3 evidence-associated genes (1 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

PathwayCohort genesFoldFDRSample cohort genes
Regulation of CDH1 mRNA translation by microRNAs11038.2×0.005MYCN
Signaling by ALK1571.0×0.005MYCN
TGFBR3 expression1456.8×0.005MYCN
Signaling by TGFBR31368.4×0.005MYCN
Signaling by TGFB family members1115.3×0.012MYCN
Regulation of PD-L1(CD274) transcription1108.8×0.012MYCN
Signaling by Receptor Tyrosine Kinases151.7×0.022MYCN
Signal Transduction110.2×0.098MYCN

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO termCohort genesFoldFDRSample cohort genes
regulation of inner ear auditory receptor cell differentiation12808.7×0.006MYCN
negative regulation of kinase activity12106.5×0.006MYCNOS
autosome genomic imprinting11203.7×0.007MYCN
negative regulation of astrocyte differentiation1766.0×0.008MYCN
positive regulation of programmed cell death1561.7×0.008MYCN
negative regulation of reactive oxygen species metabolic process1468.1×0.008MYCN
cartilage condensation1383.0×0.008MYCN
astrocyte differentiation1383.0×0.008MYCN
branching morphogenesis of an epithelial tube1366.4×0.008MYCN
positive regulation of mesenchymal cell proliferation1300.9×0.008MYCN
embryonic skeletal system morphogenesis1195.9×0.012MYCN
epithelial cell proliferation1156.0×0.012MYCN
embryonic digit morphogenesis1150.5×0.012MYCN
positive regulation of miRNA transcription1145.3×0.012MYCN
positive regulation of epithelial cell proliferation1122.1×0.014MYCN
lung development199.1×0.016MYCN
regulation of protein stability162.9×0.023MYCNOS
negative regulation of gene expression134.5×0.039MYCN
protein stabilization133.4×0.039MYCNOS
positive regulation of cell migration130.9×0.040MYCNOS
positive regulation of gene expression119.4×0.061MYCN
negative regulation of apoptotic process117.4×0.064MYCNOS
positive regulation of DNA-templated transcription114.0×0.076MYCN
positive regulation of transcription by RNA polymerase II17.4×0.135MYCN
regulation of transcription by RNA polymerase II15.8×0.164MYCN

Therapeutics

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 3

Druggability breadth: 1 of 3 evidence-associated genes (33%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

SymbolMoleculesMax phase
MYCN00
MYCNOS00
TTN-AS100

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

SymbolAssaysType breakdown
MYCN11Binding:11

Pharmacogenomics

Cohort genes with a PharmGKB record: 2; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

0 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

TierDefinitionGenesSymbols
AApproved (phase 4 drug)0
BPhased (≥1) drug, not yet approved0
CDruggable family + PDB, no drug0
DDruggable family + AlphaFold only, no drug0
EDifficult family or no structure, no drug3MYCN, MYCNOS, TTN-AS1

Undrugged target profiles

3 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

SymbolChEMBL assaysDrugged partners (top 3)
MYCN11
MYCNOS0
TTN-AS10

Clinical trials & evidence

Clinical trials

Clinical trials: 0.