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Neuroblastoma mutation landscape

A machine-readable, cohort-aware view of genomic alterations, variant provenance and therapeutic interpretation in Neuroblastoma.

Last updated 2026-09-09 · Sources: cBioPortal · Neuroblastoma (AMC Amsterdam, Nature 2012) (nbl_amc_2012), cBioPortal · Neuroblastoma (Broad, Nature 2015) (nbl_ucologne_2015), cBioPortal · Neuroblastoma (Broad, Nat Genet 2013) (nbl_broad_2013), cBioPortal · Pediatric Neuroblastoma (TARGET, 2018) (nbl_target_2018_pub), cBioPortal · Pediatric Pan-Cancer (DKFZ, Nature 2018) (pediatric_dkfz_2017), cBioPortal · Pediatric Neuroblastoma (MSK, Nat Genet 2023) (nbl_msk_2023) · Source status: PUBLIC SOURCE SNAPSHOT · Read report · JSON: /disease/neuroblastoma/mutations.json

6 cohorts, 8 alteration types, coordinates on hg19 as published. Patients are not pooled across cohorts: overlap between these studies has not been checked, so every percentage on this page divides by one named cohort and there is no total.

Mutation landscape visual

The visual repeats values present in the semantic tables below; percentages use the MSK reference cohort unless a cohort is named.

What is altered, by cohort

One row per alteration rather than per gene, because a gene has more than one. MYCN is point-mutated in 1 of 176 MSK patients and amplified in 46 of the same 176 — a single row per gene reports the first and hides the second, which is the alteration the disease is risk-stratified by. Every cell keeps its own denominator.

Alterationnbl_amc_2012nbl_ucologne_2015nbl_broad_2013nbl_target_2018_pubpediatric_dkfz_2017nbl_msk_2023
ALK SNV / small indel5.75%5/878.93%5/569.17%22/24013.59%64/4712.0%1/5016.48%29/176
ALK high-level amplification···1.69%1/59·2.27%4/176
MYCN SNV / small indel1.15%1/870%1.67%4/2401.27%6/4710%0.57%1/176
MYCN high-level amplification···18.64%11/59·26.14%46/176
ATRX SNV / small indel0%1.79%1/562.5%6/2402.76%13/4710%8.52%15/176
ATRX homozygous deletion···1.69%1/59·3.41%6/176
TERT SNV / small indel·····3.98%7/176
TERT high-level amplification···3.39%2/59·1.7%3/176
TERT homozygous deletion·····0.57%1/176
TP53 SNV / small indel0%0%0.42%1/2400.64%3/4712.0%1/501.14%2/176
TP53 high-level amplification·····0.57%1/176
TP53 homozygous deletion·····0.57%1/176
PHOX2B SNV / small indel0%0%0%0.42%2/4712.0%1/501.14%2/176
BARD1 SNV / small indel0%0%0%0.21%1/4710%0%
BARD1 homozygous deletion···1.69%1/59··
1p36 deletion chromosome arm···30.51%18/59··
17q gain chromosome arm···83.05%49/59··

observed — shade scales with frequency assayed, none found not assayed here cohort not evaluable

A measured zero and an unmeasured one are different facts, so they are drawn differently. TERT is the worked example: MSK-IMPACT tiles the promoter and the whole-genome studies report coding variants only. The last two rows are chromosome arms — 17q gain is the commonest structural change in neuroblastoma and has no gene row at all; both are gene-level proxies over TARGET's GISTIC profile, not segment calls, and both divide by its 59 profiled patients.

Assay coverage by cohort

Patients with at least one mutation call, over the patients the cohort lists. This is why one cohort carries no frequencies: a roster the profile never reached is not a denominator.

nbl_amc_2012 73/87
nbl_ucologne_2015 56/56
nbl_broad_2013 237/240
nbl_target_2018_pub 414/471
pediatric_dkfz_2017 49/50
nbl_msk_2023 129/176

Amber marks a cohort excluded from frequency tables. The measure understates panels: a patient with no mutation in the panel's genes is a real negative and still lowers it.

Data modality coverage

SNV / small indel6/6
Copy-number alteration2/6
Structural variant2/6
Fusion-annotated SV2/6

Counts source cohorts with a connected public profile, not pooled patients.

Answer block

6 public Neuroblastoma cohorts are connected from cBioPortal: AMC 2012, Cologne 2015, Broad 2013, TARGET, DKFZ 2017, MSK 2023.

Each gene is reported by its largest alteration in MSK 2023, whichever kind that is: MYCN 46/176 (26.14%, high-level amplification), ALK 29/176 (16.48%, SNV / small indel), ATRX 15/176 (8.52%, SNV / small indel), TERT 7/176 (3.98%, SNV / small indel), TP53 2/176 (1.14%, SNV / small indel), PHOX2B 2/176 (1.14%, SNV / small indel). MSK-IMPACT is a targeted panel applied to a clinically selected series, so these run higher than the genome-wide cohorts.

ALK ranges from 2.0% (DKFZ 2017) to 16.48% (MSK 2023) across the 6 evaluable cohorts; these values are not pooled.

1p36 deletion is counted in 18 of 59 TARGET patients with a GISTIC profile (30.51%): more than half of 6 genes spanning 1p36 at GISTIC -1 or beyond. That is the hemizygous threshold, not the ±2 used by the gene-level copy-number rows below; read at ±2 this count would be 3. It is a gene-level proxy, not a segment call: no study in this snapshot exposes copy-number segments.

17q gain is counted in 49 of 59 TARGET patients with a GISTIC profile (83.05%): more than half of 6 genes spanning 17q at GISTIC +1 or beyond. That is the hemizygous threshold, not the ±2 used by the gene-level copy-number rows below; read at ±2 this count would be 1. It is a gene-level proxy, not a segment call: no study in this snapshot exposes copy-number segments.

Unknown, not assayed, not observed and zero are kept as separate states; every numeric value retains a source ID, retrieval date and counting unit.

Evidence boundary: no active-trial, approved-drug, absent-alteration or functional structural-variant conclusion is asserted by this snapshot. Those statements require a separate searched source and retrieval date.

Key findings

ALK is observed in all 6 evaluable cohorts, 2.0% to 16.48%; the MSK reference frequency is 16.48% on a targeted panel.
Numerator: 29 · Denominator: 176 · Frequency: 16.48 · Cohorts: 5 · Confidence: moderate · Source: nbl_msk_2023 · Retrieved: 2026-09-09

TERT is reported only by MSK-IMPACT, which tiles the promoter; the whole-genome studies report coding variants and never called it, so their zeros are missing coverage rather than absence.
Numerator: 7 · Denominator: 176 · Frequency: 3.98 · Cohorts: 1 · Confidence: moderate · Source: nbl_msk_2023 · Retrieved: 2026-09-09

ATRX is observed in 4 of the 6 evaluable cohorts and reaches 8.52% in the MSK reference profile.
Numerator: 15 · Denominator: 176 · Frequency: 8.52 · Cohorts: 3 · Confidence: moderate · Source: nbl_msk_2023 · Retrieved: 2026-09-09

MYCN mutation calls are uncommon; separate CNA profiles show high-level amplification in 46/176 MSK patients and 11/59 TARGET patients.
Numerator: 1 · Denominator: 176 · Frequency: 0.57 · Cohorts: 4 · Confidence: moderate · Source: nbl_msk_2023 · Retrieved: 2026-09-09

MYCN high-level amplification is observed in 46/176 MSK patients (26.14%) and 11/59 TARGET patients (18.64%) with CNA data.
Numerator: 46 · Denominator: 176 · Frequency: 26.14 · Cohorts: 2 · Confidence: moderate · Source: nbl_msk_2023_cna; nbl_target_2018_pub_gistic · Retrieved: 2026-09-09

Structural-variant profiles contain 79 records in Broad 2013 and 16 in MSK 2023; the Broad rows divide by the 19 patients with an SV profile, not by its 240 patients.
Numerator: null · Denominator: null · Frequency: null · Cohorts: 2 · Confidence: moderate · Source: nbl_broad_2013_structural_variants; nbl_msk_2023_structural_variants · Retrieved: 2026-09-09

Mutation frequency table

Frequencies vary by cohort, assay, age group, disease subtype and sequencing methodology. Headline values use the MSK 2023 reference cohort (176 unique patients); the cohort table keeps every denominator separate.

Gene-level observations · null values are explicit missingness
GeneChromosomeAlteration typeAltered patients/samplesTested/evaluableFrequencyNumber of cohortsMajor variants/eventsEvidence confidenceSource
ALKchr2SNV, small indel 29 observed176 observed 16.48% observed6 F1174L (13 patients), R1275Q (6 patients), F1245V (3 patients)moderatenbl_msk_2023
MYCNchr2SNV 1 observed176 observed 0.57% observed4 P44L (1 patient; 4 samples)moderatenbl_msk_2023
ATRXchrXSNV, small indel 15 observed176 observed 8.52% observed4 P663Yfs*10 (1 patient), A1690D (1 patient), R1739Hfs*8 (1 patient)moderatenbl_msk_2023
TERTchr5SNV, promoter variant 7 observed176 observed 3.98% observed1 Promoter (5 patients), R466W (1 patient), S838Y (1 patient)moderatenbl_msk_2023
TP53chr17SNV 2 observed176 observed 1.14% observed4 Y234* (1 patient; 2 samples), G154V (1 patient)moderatenbl_msk_2023
PHOX2Bchr4small indel 2 observed176 observed 1.14% observed3 G272Rfs*88 (1 patient), P290Sfs*70 (1 patient)moderatenbl_msk_2023
BARD1chr2SNV, small indel 0 not_observed176 observed 0.0% not_observed1 none observed in the selected mutation profilesmoderatenbl_msk_2023

Cohort breakdown

Cohorts remain separate until overlap, assay coverage and counting unit are documented.

CohortSource / accessionPatientsSamplesSubtypeAssay / sequencingBuildAlterationsCoveragePrimary / metastaticAge groupRetrievedLicence
Neuroblastoma (AMC Amsterdam, Nature 2012)cBioPortal
nbl_amc_2012
87 observed87 observedPrimary neuroblastomaWhole-genome sequencing
WGS tumor/normal pairs
hg19SNV, small indelGenome-wide mutation profile; no CNA/SV profile connectedPrimary tumorsPediatric study cohort2026-09-09cBioPortal public study; original study terms apply
Neuroblastoma (Broad, Nature 2015)cBioPortal
nbl_ucologne_2015
56 observed56 observedNeuroblastomaWhole-genome sequencing
WGS tumor/normal pairs
hg19SNV, small indelGenome-wide mutation profile; no CNA/SV profile connectedTumor/normal pairs; status not harmonizedPediatric study cohort2026-09-09cBioPortal public study; original study terms apply
Neuroblastoma (Broad, Nat Genet 2013)cBioPortal
nbl_broad_2013
240 observed240 observedHigh-risk neuroblastomaWhole-genome and whole-exome sequencing
WGS/WES tumor/normal pairs
hg19SNV, small indel, structural variantGenome-wide mutation profile plus structural-variant profileTumor/normal pairs; status not harmonizedPediatric study cohort2026-09-09cBioPortal public study; original study terms apply
Pediatric Neuroblastoma (TARGET, 2018)cBioPortal
nbl_target_2018_pub
471 observed533 observedPediatric neuroblastomaWhole-genome or whole-exome sequencing
WGS/WES
hg19SNV, small indel, copy-number alterationMutation calls from the 2025 GDC reprocessing (nbl_target_gdc_mutations, 414/471 samples called); copy-number from the 2018 release's GISTIC profile, which the GDC study does not carryTumor/normal pairs; status not harmonizedPediatric study cohort2026-09-09TARGET public data; OCG TARGET using-data and publication guidelines apply
Pediatric Pan-Cancer (DKFZ, Nature 2018)cBioPortal
pediatric_dkfz_2017
50 observed59 observedNeuroblastoma subset of a pan-cancer studyWhole-genome and whole-exome sequencing
WGS/WES tumor/normal pairs
hg19SNV, small indelGenome-wide mutation profile; no copy-number or structural-variant profile is connected for this studyPrimary pediatric tumours; status not harmonizedPediatric study cohort2026-09-09cBioPortal public study; original study terms apply
Pediatric Neuroblastoma (MSK, Nat Genet 2023)cBioPortal
nbl_msk_2023
176 observed223 observedPediatric neuroblastomaTargeted DNA sequencing
MSK-IMPACT 341/410/468/505; one HEME-400 profile
hg19SNV, small indel, copy-number alteration, structural variantPanel coverage retained per sample; all seven genes plus GISTIC CNA/SV profilesTumor/normal pairs; primary/relapse not harmonizedPediatric study cohort2026-09-09cBioPortal public study; original study terms apply

Copy-number alterations

Discrete GISTIC calls are shown for the TARGET 2018 and MSK 2023 profiles. Code 2 is high-level amplification and code −2 is homozygous deletion; patient denominators are profile-specific.

GeneEventObserved patientsTested patientsFrequencyCohortSource profile
MYCNhigh-level amplification115918.64%nbl_target_2018_pubnbl_target_2018_pub_gistic
ALKhigh-level amplification1591.69%nbl_target_2018_pubnbl_target_2018_pub_gistic
ATRXhomozygous deletion1591.69%nbl_target_2018_pubnbl_target_2018_pub_gistic
TERThigh-level amplification2593.39%nbl_target_2018_pubnbl_target_2018_pub_gistic
BARD1homozygous deletion1591.69%nbl_target_2018_pubnbl_target_2018_pub_gistic
MYCNhigh-level amplification4617626.14%nbl_msk_2023nbl_msk_2023_cna
ALKhigh-level amplification41762.27%nbl_msk_2023nbl_msk_2023_cna
ATRXhomozygous deletion61763.41%nbl_msk_2023nbl_msk_2023_cna
TERThigh-level amplification31761.7%nbl_msk_2023nbl_msk_2023_cna
TERThomozygous deletion11760.57%nbl_msk_2023nbl_msk_2023_cna
TP53high-level amplification11760.57%nbl_msk_2023nbl_msk_2023_cna
TP53homozygous deletion11760.57%nbl_msk_2023nbl_msk_2023_cna

Structural variants and fusion-annotated events

Broad 2013 and MSK 2023 expose structural-variant profiles. Fusion labels are preserved from the source annotation; they do not establish a functional driver or therapeutic sensitivity.

TypeGene / partnerEventObserved patientsTested patientsFrequencyCohortCoordinates / details
structural_variantGenome-wide79 SV records161984.21%nbl_broad_201379 records across 16 patients; 30 of them name a fusion in eventInfo
fusionGenome-wideFusion-annotated SV records131968.42%nbl_broad_201330 rows across 13 patients, counted as rows whose cBioPortal eventInfo names a fusion, not rows with two named partner genes -- those are 75 and 26 respectively in Broad 2013. Functional impact is not established: variantClass is NA on every row.
fusionMYCN · GULP1MYCN–GULP1 fusion1195.26%nbl_broad_2013Fusion annotation from the Broad 2013 SV profile; breakpoints were not supplied
structural_variantGenome-wide16 SV records111766.25%nbl_msk_202316 records across 11 patients in the MSK SV profile
fusionGenome-wideFusion-annotated SV records31761.7%nbl_msk_20234 rows across 3 patients, counted as rows whose cBioPortal eventInfo names a fusion, not rows with two named partner genes -- those are 75 and 26 respectively in Broad 2013. Functional impact is not established.
structural_variantALK · ALKIntragenic ALK duplication11760.57%nbl_msk_2023GRCh37:2:29448689–2:29572365 · GRCh37:2:29448689–2:29572365; predicted in-frame duplication
fusionARID1A · SFNARID1A–SFN transcript fusion11760.57%nbl_msk_2023Deletion-class SV with transcript-fusion annotation
fusionATRX · ATRXATRX antisense fusion11760.57%nbl_msk_2023GRCh37:X:76909487–X:77030840 · GRCh37:X:76909487–X:77030840; intragenic inversion
fusionVAX2 · TMEM127VAX2–TMEM127 protein fusion11760.57%nbl_msk_2023Inversion-class SV with mid-exon protein-fusion annotation

Chromosome-wise event summary

This rollup links the seven selected genes and events anchored to those genes to their source chromosomes. It is useful for orientation and filtering; it is not a genome-wide mutation burden or a complete cytogenetic profile.

ChromosomeSelected genesMutation recordsGene-patient observationsRepresentative variantsCNA event rowsSV event rowsFusion rowsSource scope
chr2ALK, MYCN, BARD11491394511Selected 7 genes; mutation rows summed across 6 cohorts; patients are not pooled.
nbl_amc_2012, nbl_ucologne_2015, nbl_broad_2013, nbl_target_2018_pub, pediatric_dkfz_2017, nbl_msk_2023
chr4PHOX2B552000Selected 7 genes; mutation rows summed across 6 cohorts; patients are not pooled.
nbl_amc_2012, nbl_ucologne_2015, nbl_broad_2013, nbl_target_2018_pub, pediatric_dkfz_2017, nbl_msk_2023
chr5TERT773300Selected 7 genes; mutation rows summed across 6 cohorts; patients are not pooled.
nbl_amc_2012, nbl_ucologne_2015, nbl_broad_2013, nbl_target_2018_pub, pediatric_dkfz_2017, nbl_msk_2023
chr17TP53872200Selected 7 genes; mutation rows summed across 6 cohorts; patients are not pooled.
nbl_amc_2012, nbl_ucologne_2015, nbl_broad_2013, nbl_target_2018_pub, pediatric_dkfz_2017, nbl_msk_2023
chrXATRX45353201Selected 7 genes; mutation rows summed across 6 cohorts; patients are not pooled.
nbl_amc_2012, nbl_ucologne_2015, nbl_broad_2013, nbl_target_2018_pub, pediatric_dkfz_2017, nbl_msk_2023

Gene details

These sections are server-rendered so their content is available to crawlers and retrieval systems without client-side interaction.

ALK

Chromosomechr2

Observed alteration typesSNV, small indel

Recurrent variants/eventsF1174L (13 patients), R1275Q (6 patients), F1245V (3 patients)

Altered / tested denominator29 / 176 (observed / observed)

Cohort-specific frequenciesavailable below

Disease relevanceALK mutations are observed in every cohort whose calls cover its roster — four of the five here; TARGET 2018 is not evaluable. Biological and clinical interpretation remains source-specific.

Druggability / targetabilityMutation observations only; targetability, approved drugs and trial eligibility require linked clinical evidence.

Approved drugs in other diseasesnot curated in snapshot

Neuroblastoma trialsnot searched · no negative claim

Cross-disease activitynot connected

Related BioTransfer tools/pagesTarget–Disease Explorer · Disease briefings

Limitations: Headline altered/tested/frequency values use the MSK 2023 mutation profile (176 unique patients) as the reference cohort. Cohort rows are not pooled; zero means no call in the selected profile and is not a biological absence claim. Drug approvals and trials are not curated in this snapshot; genomic event records are shown in their dedicated tables with event-specific denominators.

MYCN

Chromosomechr2

Observed alteration typesSNV

Recurrent variants/eventsP44L (1 patient; 4 samples)

Altered / tested denominator1 / 176 (observed / observed)

Cohort-specific frequenciesavailable below

Disease relevanceMYCN mutation calls are uncommon in these profiles; copy-number amplification is represented separately in the CNA event table.

Druggability / targetabilityMutation observations only; targetability, approved drugs and trial eligibility require linked clinical evidence.

Approved drugs in other diseasesnot curated in snapshot

Neuroblastoma trialsnot searched · no negative claim

Cross-disease activitynot connected

Related BioTransfer tools/pagesTarget–Disease Explorer · Disease briefings

Limitations: Headline altered/tested/frequency values use the MSK 2023 mutation profile (176 unique patients) as the reference cohort. Cohort rows are not pooled; zero means no call in the selected profile and is not a biological absence claim. Drug approvals and trials are not curated in this snapshot; genomic event records are shown in their dedicated tables with event-specific denominators.

ATRX

ChromosomechrX

Observed alteration typesSNV, small indel

Recurrent variants/eventsP663Yfs*10 (1 patient), A1690D (1 patient), R1739Hfs*8 (1 patient)

Altered / tested denominator15 / 176 (observed / observed)

Cohort-specific frequenciesavailable below

Disease relevanceATRX mutations are observed in the Broad 2013, Cologne 2015 and MSK 2023 profiles; this page does not infer a phenotype or prognosis.

Druggability / targetabilityMutation observations only; targetability, approved drugs and trial eligibility require linked clinical evidence.

Approved drugs in other diseasesnot curated in snapshot

Neuroblastoma trialsnot searched · no negative claim

Cross-disease activitynot connected

Related BioTransfer tools/pagesTarget–Disease Explorer · Disease briefings

Limitations: Headline altered/tested/frequency values use the MSK 2023 mutation profile (176 unique patients) as the reference cohort. Cohort rows are not pooled; zero means no call in the selected profile and is not a biological absence claim. Drug approvals and trials are not curated in this snapshot; genomic event records are shown in their dedicated tables with event-specific denominators.

TERT

Chromosomechr5

Observed alteration typesSNV, promoter variant

Recurrent variants/eventsPromoter (5 patients), R466W (1 patient), S838Y (1 patient)

Altered / tested denominator7 / 176 (observed / observed)

Cohort-specific frequenciesavailable below

Disease relevanceTERT promoter and coding mutations are observed in the MSK profile; promoter calls are retained as a separate event type.

Druggability / targetabilityMutation observations only; targetability, approved drugs and trial eligibility require linked clinical evidence.

Approved drugs in other diseasesnot curated in snapshot

Neuroblastoma trialsnot searched · no negative claim

Cross-disease activitynot connected

Related BioTransfer tools/pagesTarget–Disease Explorer · Disease briefings

Limitations: Headline altered/tested/frequency values use the MSK 2023 mutation profile (176 unique patients) as the reference cohort. Cohort rows are not pooled; zero means no call in the selected profile and is not a biological absence claim. Drug approvals and trials are not curated in this snapshot; genomic event records are shown in their dedicated tables with event-specific denominators.

TP53

Chromosomechr17

Observed alteration typesSNV

Recurrent variants/eventsY234* (1 patient; 2 samples), G154V (1 patient)

Altered / tested denominator2 / 176 (observed / observed)

Cohort-specific frequenciesavailable below

Disease relevanceTP53 mutations are observed in two source cohorts; the snapshot does not classify disease subtype or treatment response.

Druggability / targetabilityMutation observations only; targetability, approved drugs and trial eligibility require linked clinical evidence.

Approved drugs in other diseasesnot curated in snapshot

Neuroblastoma trialsnot searched · no negative claim

Cross-disease activitynot connected

Related BioTransfer tools/pagesTarget–Disease Explorer · Disease briefings

Limitations: Headline altered/tested/frequency values use the MSK 2023 mutation profile (176 unique patients) as the reference cohort. Cohort rows are not pooled; zero means no call in the selected profile and is not a biological absence claim. Drug approvals and trials are not curated in this snapshot; genomic event records are shown in their dedicated tables with event-specific denominators.

PHOX2B

Chromosomechr4

Observed alteration typessmall indel

Recurrent variants/eventsG272Rfs*88 (1 patient), P290Sfs*70 (1 patient)

Altered / tested denominator2 / 176 (observed / observed)

Cohort-specific frequenciesavailable below

Disease relevancePHOX2B mutation calls are observed in the MSK profile; germline susceptibility and tumor mutation evidence must remain separate.

Druggability / targetabilityMutation observations only; targetability, approved drugs and trial eligibility require linked clinical evidence.

Approved drugs in other diseasesnot curated in snapshot

Neuroblastoma trialsnot searched · no negative claim

Cross-disease activitynot connected

Related BioTransfer tools/pagesTarget–Disease Explorer · Disease briefings

Limitations: Headline altered/tested/frequency values use the MSK 2023 mutation profile (176 unique patients) as the reference cohort. Cohort rows are not pooled; zero means no call in the selected profile and is not a biological absence claim. Drug approvals and trials are not curated in this snapshot; genomic event records are shown in their dedicated tables with event-specific denominators.

BARD1

Chromosomechr2

Observed alteration typesSNV, small indel

Recurrent variants/eventsnone observed in the selected mutation profiles

Altered / tested denominator0 / 176 (not_observed / observed)

Cohort-specific frequenciesavailable below

Disease relevanceNo BARD1 mutation call is present in the selected public mutation profiles; this is not evidence against germline risk.

Druggability / targetabilityMutation observations only; targetability, approved drugs and trial eligibility require linked clinical evidence.

Approved drugs in other diseasesnot curated in snapshot

Neuroblastoma trialsnot searched · no negative claim

Cross-disease activitynot connected

Related BioTransfer tools/pagesTarget–Disease Explorer · Disease briefings

Limitations: Headline altered/tested/frequency values use the MSK 2023 mutation profile (176 unique patients) as the reference cohort. Cohort rows are not pooled; zero means no call in the selected profile and is not a biological absence claim. Drug approvals and trials are not curated in this snapshot; genomic event records are shown in their dedicated tables with event-specific denominators.

Cohort-aware frequencies

Each row is calculated from unique patients in that study's sequenced sample list. The range is descriptive; no pooled estimate is shown because cross-study overlap and assay comparability have not been checked.

GeneObserved rangeSource cohort frequencies
ALK2.0–16.48%nbl_amc_2012: 5/87 (5.75%) · nbl_ucologne_2015: 5/56 (8.93%) · nbl_broad_2013: 22/240 (9.17%) · nbl_target_2018_pub: 64/471 (13.59%) · pediatric_dkfz_2017: 1/50 (2.0%) · nbl_msk_2023: 29/176 (16.48%)
MYCN0.0–1.67%nbl_amc_2012: 1/87 (1.15%) · nbl_ucologne_2015: 0/56 (0.0%) · nbl_broad_2013: 4/240 (1.67%) · nbl_target_2018_pub: 6/471 (1.27%) · pediatric_dkfz_2017: 0/50 (0.0%) · nbl_msk_2023: 1/176 (0.57%)
ATRX0.0–8.52%nbl_amc_2012: 0/87 (0.0%) · nbl_ucologne_2015: 1/56 (1.79%) · nbl_broad_2013: 6/240 (2.5%) · nbl_target_2018_pub: 13/471 (2.76%) · pediatric_dkfz_2017: 0/50 (0.0%) · nbl_msk_2023: 15/176 (8.52%)
TERT3.98–3.98%nbl_amc_2012: None/87 (None%) · nbl_ucologne_2015: None/56 (None%) · nbl_broad_2013: None/240 (None%) · nbl_target_2018_pub: None/471 (None%) · pediatric_dkfz_2017: None/50 (None%) · nbl_msk_2023: 7/176 (3.98%)
TP530.0–2.0%nbl_amc_2012: 0/87 (0.0%) · nbl_ucologne_2015: 0/56 (0.0%) · nbl_broad_2013: 1/240 (0.42%) · nbl_target_2018_pub: 3/471 (0.64%) · pediatric_dkfz_2017: 1/50 (2.0%) · nbl_msk_2023: 2/176 (1.14%)
PHOX2B0.0–2.0%nbl_amc_2012: 0/87 (0.0%) · nbl_ucologne_2015: 0/56 (0.0%) · nbl_broad_2013: 0/240 (0.0%) · nbl_target_2018_pub: 2/471 (0.42%) · pediatric_dkfz_2017: 1/50 (2.0%) · nbl_msk_2023: 2/176 (1.14%)
BARD10.0–0.21%nbl_amc_2012: 0/87 (0.0%) · nbl_ucologne_2015: 0/56 (0.0%) · nbl_broad_2013: 0/240 (0.0%) · nbl_target_2018_pub: 1/471 (0.21%) · pediatric_dkfz_2017: 0/50 (0.0%) · nbl_msk_2023: 0/176 (0.0%)

Variant table

Representative SNV and small-indel records from the MSK 2023 mutation profile. Transcript and HGVS.c remain null where cBioPortal did not provide them; source hg19 coordinates are retained.

GeneCoordinateRefAltTranscriptHGVS c.HGVS p.ConsequenceClassObservedTestedDiseasesCohortsSource IDs
ALK2:29443695GCnullnullF1174LMissense_MutationSNV13 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
ALK2:29432664CTnullnullR1275QMissense_MutationSNV6 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
ALK2:29436860ACnullnullF1245VMissense_MutationSNV3 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
MYCN2:16082317CTnullnullP44LMissense_MutationSNV1 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
ATRXX:76938761-76938762-TGTAnullnullP663Yfs*10Frame_Shift_Inssmall indel1 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
ATRXX:76888760GTnullnullA1690DMissense_MutationSNV1 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
ATRXX:76875919C-nullnullR1739Hfs*8Frame_Shift_Delsmall indel1 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
TERT5:1295228GAnullnullPromoter5'Flankregulatory SNV5 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
TERT5:1293605GAnullnullR466WMissense_MutationSNV1 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
TERT5:1268704GTnullnullS838YMissense_MutationSNV1 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
TP5317:7577579GTnullnullY234*Nonsense_MutationSNV1 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
TP5317:7578469CAnullnullG154VMissense_MutationSNV1 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
PHOX2B4:41747955-41747956-TnullnullG272Rfs*88Frame_Shift_Inssmall indel1 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023
PHOX2B4:41747902-41747903-CnullnullP290Sfs*70Frame_Shift_Inssmall indel1 patients176 patientsNeuroblastomanbl_msk_2023nbl_msk_2023

Mutation types supported

SNVsmall indelcopy-number gainamplificationdeletionstructural variantrearrangementfusion

SNV/small-indel calls are loaded across all 6 cohorts; CNA is loaded for TARGET/MSK, and SV/fusion profiles for Broad/MSK. The schema remains extensible for signatures and additional event-specific annotations.

Related intelligence

Each normalized mutation can later connect through BioTransfer's graph:

Mutation → Gene → Target → Drug → Approval → Disease → Clinical trial → Dataset → Publication → Funding

Approval in another disease, a neuroblastoma trial and established neuroblastoma actionability are separate evidence categories. The local fixture does not infer one from another.

Open Target–Disease Explorer for connected drug and trial records when available.

Research gaps

Assay coverage
Extend coverage to cohorts and genomic profiles not yet connected, with richer event annotations and event-specific tested denominators.

Clinical evidence
Link approved drugs, resistance evidence and active Neuroblastoma trials through separately dated evidence sources.

Cohort harmonization
Check cross-study patient overlap and harmonize primary/relapse, age and risk-group definitions before any pooled estimate.

Variant normalization
Add transcript and HGVS.c mappings while retaining the original hg19 coordinate and raw cBioPortal row.

Limitations

How a machine should read this page

  1. Denominators: frequency is calculated only from confirmed evaluable patients or samples, using the same counting unit and event-specific assay coverage.
  2. Missing values: unknown is represented as null with a status such as unknown, not_assayed or unavailable; it is never silently converted to zero.
  3. Variant normalization: source build is retained; GRCh37/GRCh38 liftover, left normalization, transcript mapping and HGVS representation are recorded in provenance. Raw records are never overwritten.
  4. Patient/sample deduplication: multiple samples from one patient require a documented counting rule; duplicate identifiers are removed only when identity is confirmed.
  5. Cross-cohort pooling: cohort frequencies are shown separately. Pooling requires documented overlap checks, compatible disease/age/subtype definitions and comparable assay coverage.
  6. Provenance: every derived value retains source, source record ID, cohort, retrieval date, source version, processing version, normalization method, genome build and licence metadata.
  7. Negative evidence: use explicit statements only when supported by a searched source and retrieval date: “No active trial identified”, “No approved drug”, “Tested denominator unavailable” or “Structural-variant data unavailable”.

Machine endpoints: full landscape · genes · cohorts.

BioTransfer local development release. This public-source snapshot is intended for review before deployment. Source adapters can populate the same contract for glioblastoma, AML, melanoma, pancreatic, lung, breast, ovarian and other cancers without changing the page model.