OncoSignal
Indications / Non-small cell lung cancer · Breast cancer · Pancreatic cancer · Colorectal cancer · Acute myeloid leukemia · Chronic myeloid leukemia · Multiple myeloma · Glioblastoma · Glioma · Neuroblastoma · Medulloblastoma · Diffuse midline glioma (DIPG) · Ependymoma · Atypical teratoid rhabdoid tumour (AT/RT) · Childhood acute lymphoblastic leukemia · Ewing sarcoma · Osteosarcoma · Rhabdomyosarcoma

Rhabdomyosarcoma

The commonest soft-tissue sarcoma of childhood; PAX3::FOXO1 fusion-positive and metastatic disease do worst.

Live from Open Targets Platform, fetched 2026-11-11 15:26 UTC. Every row links to its source record.
Targets linked to rhabdomyosarcoma
2,164
the top 50 by overall evidence are shown
With inherited (germline) evidence
15
of the top 50, score above 0.1
Strong genetics, few drugs
33
genetic evidence 0.5 or more, drug evidence under 0.5
33 of the top 50 rhabdomyosarcoma targets have strong human genetic evidence but little drug evidence so far

Each dot is one target. Higher means stronger genetic evidence in people (inherited variants or tumour mutations, whichever is stronger). Further right means more evidence from drugs already in trials or approved.

Has inherited (germline) evidence above 0.1Tumour (somatic) or other evidence only
000.250.250.50.50.750.7511Drug evidence (trials and approvals) →Human genetic evidence →Genetics strong, few drugs: open betsGenetics strong, drugs in the clinicDrugs in the clinic, weaker geneticsDICER1: genetic evidence 0.90 (germline 0.90, somatic 0.80), drug evidence 0.00PAX3: genetic evidence 0.91 (germline 0.75, somatic 0.91), drug evidence 0.00TP53: genetic evidence 0.86 (germline 0.86, somatic 0.68), drug evidence 0.00PAX7: genetic evidence 0.90 (germline 0.52, somatic 0.90), drug evidence 0.00NF1: genetic evidence 0.84 (germline 0.84, somatic 0.63), drug evidence 0.00FGFR4: genetic evidence 0.79 (germline 0.00, somatic 0.79), drug evidence 0.00FOXO1: genetic evidence 0.91 (germline 0.00, somatic 0.91), drug evidence 0.00BRCA2: genetic evidence 0.82 (germline 0.82, somatic 0.57), drug evidence 0.00MYOD1: genetic evidence 0.83 (germline 0.00, somatic 0.83), drug evidence 0.00NBN: genetic evidence 0.38 (germline 0.00, somatic 0.38), drug evidence 0.00HRAS: genetic evidence 0.68 (germline 0.68, somatic 0.64), drug evidence 0.00BRCA1: genetic evidence 0.70 (germline 0.70, somatic 0.57), drug evidence 0.00EWSR1: genetic evidence 0.83 (germline 0.00, somatic 0.83), drug evidence 0.00FLI1: genetic evidence 0.83 (germline 0.00, somatic 0.83), drug evidence 0.00NRAS: genetic evidence 0.80 (germline 0.00, somatic 0.80), drug evidence 0.00WT1: genetic evidence 0.79 (germline 0.00, somatic 0.79), drug evidence 0.00SLC67A1: genetic evidence 0.55 (germline 0.00, somatic 0.55), drug evidence 0.00ERG: genetic evidence 0.77 (germline 0.00, somatic 0.77), drug evidence 0.00PTEN: genetic evidence 0.62 (germline 0.55, somatic 0.62), drug evidence 0.00SDHA: genetic evidence 0.68 (germline 0.68, somatic 0.30), drug evidence 0.00SH2B3: genetic evidence 0.76 (germline 0.00, somatic 0.76), drug evidence 0.00ALK: genetic evidence 0.62 (germline 0.43, somatic 0.62), drug evidence 0.12PIK3CA: genetic evidence 0.66 (germline 0.00, somatic 0.66), drug evidence 0.00PMS2: genetic evidence 0.57 (germline 0.55, somatic 0.57), drug evidence 0.00ASPSCR1: genetic evidence 0.72 (germline 0.00, somatic 0.72), drug evidence 0.00FBXW7: genetic evidence 0.62 (germline 0.00, somatic 0.62), drug evidence 0.00TFE3: genetic evidence 0.72 (germline 0.00, somatic 0.72), drug evidence 0.00SFRP4: genetic evidence 0.72 (germline 0.00, somatic 0.72), drug evidence 0.00LRP1B: genetic evidence 0.56 (germline 0.00, somatic 0.56), drug evidence 0.00MSH6: genetic evidence 0.55 (germline 0.55, somatic 0.46), drug evidence 0.00CTNNB1: genetic evidence 0.55 (germline 0.00, somatic 0.55), drug evidence 0.00TOP1: genetic evidence 0.15 (germline 0.00, somatic 0.15), drug evidence 0.65SSX1: genetic evidence 0.68 (germline 0.00, somatic 0.68), drug evidence 0.00TUBB: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68PTCH1: genetic evidence 0.57 (germline 0.43, somatic 0.57), drug evidence 0.00TUBB3: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68FBXO11: genetic evidence 0.55 (germline 0.55, somatic 0.53), drug evidence 0.00TUBA3C: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB8: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBA1C: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB4A: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBA3E: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB1: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB6: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB2A: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB4B: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBB2B: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBA1A: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBA4A: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68TUBA1B: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.68DICER1NF1FGFR4NBNHRASSLC67A1PTENALKTOP1

Scores run from 0 to 1 and are Open Targets' summary of how much evidence exists, not the chance a drug will work. The dividing lines sit at 0.5. Hover a dot for its numbers, click it for the target's page. Source: Open Targets Platform, fetched live.

Where the genetics points and the pipeline has not gone yet

The 33 targets in the top-left of the chart, strongest genetic evidence first. Click one for its drugs, tractability and other cancers.

FOXO1 0.91PAX3 0.91DICER1 0.90PAX7 0.90TP53 0.86PedsNF1 0.84MYOD1 0.83EWSR1 0.83FLI1 0.83BRCA2 0.82NRAS 0.80WT1 0.79PedsFGFR4 0.79ERG 0.77SH2B3 0.76PedsASPSCR1 0.72TFE3 0.72SFRP4 0.72BRCA1 0.70HRAS 0.68SDHA 0.68SSX1 0.68PIK3CA 0.66PedsPTEN 0.62PedsALK 0.62PedsFBXW7 0.62PMS2 0.57PTCH1 0.57LRP1B 0.56CTNNB1 0.55PedsSLC67A1 0.55MSH6 0.55FBXO11 0.55

All 50 targets, by type of evidence

Ranked by overall score for rhabdomyosarcoma on the Open Targets Platform. Darker cells mean stronger evidence of that type.

Peds RMTLmarks the 7 of these targets on the FDA's Pediatric Relevant Molecular Target List: a drug against one of them can be required to be studied in children under the RACE for Children Act.

TargetOverallGermline geneticsSomaticLiteraturegenetic literaturePathwaysAnimal modelsClinical
DICER1
dicer 1, ribonuclease III
0.780.900.80
PAX3
paired box 3
0.740.750.91
TP53Peds RMTL
tumor protein p53
0.710.860.68
PAX7
paired box 7
0.680.520.90
NF1
neurofibromin 1
0.650.840.63
FGFR4
fibroblast growth factor receptor 4
0.65–0.79
FOXO1
forkhead box O1
0.62–0.91
BRCA2
BRCA2 DNA repair associated
0.590.820.57
MYOD1
myogenic differentiation 1
0.57–0.83
NBN
nibrin
0.54–0.38
HRAS
HRas proto-oncogene, GTPase
0.540.680.64
BRCA1
BRCA1 DNA repair associated
0.510.700.57
EWSR1
EWS RNA binding protein 1
0.51–0.83
FLI1
Fli-1 proto-oncogene, ETS transcription factor
0.51–0.83
NRAS
NRAS proto-oncogene, GTPase
0.49–0.80
WT1Peds RMTL
WT1 transcription factor
0.49–0.79
SLC67A1
solute carrier family 67 member 1
0.47–0.55
ERG
ETS transcription factor ERG
0.47–0.77
PTENPeds RMTL
phosphatase and tensin homolog
0.470.550.62
SDHA
succinate dehydrogenase complex flavoprotein subunit A
0.460.680.30
SH2B3Peds RMTL
SH2B adaptor protein 3
0.46–0.76
ALKPeds RMTL
ALK receptor tyrosine kinase
0.450.430.62
PIK3CAPeds RMTL
phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha
0.45–0.66
PMS2
PMS1 homolog 2, mismatch repair system component
0.450.550.57
ASPSCR1
ASPSCR1 tether for SLC2A4, UBX domain containing
0.44–0.72
FBXW7
F-box and WD repeat domain containing 7
0.44–0.62
TFE3
transcription factor binding to IGHM enhancer 3
0.44–0.72
SFRP4
secreted frizzled related protein 4
0.44–0.72
LRP1B
LDL receptor related protein 1B
0.43–0.56
MSH6
mutS homolog 6
0.420.550.46
CTNNB1Peds RMTL
catenin beta 1
0.42–0.55
TOP1
DNA topoisomerase I
0.42–0.15
SSX1
SSX family member 1
0.42–0.68
TUBB
tubulin beta class I
0.42––
PTCH1
patched 1
0.410.430.57
TUBB3
tubulin beta 3 class III
0.41––
FBXO11
F-box protein 11
0.410.550.53
TUBA3C
tubulin alpha 3c
0.41––
TUBB8
tubulin beta 8 class VIII
0.41––
TUBA1C
tubulin alpha 1c
0.41––
TUBB4A
tubulin beta 4A class IVa
0.41––
TUBA3E
tubulin alpha 3e
0.41––
TUBB1
tubulin beta 1 class VI
0.41––
TUBB6
tubulin beta 6 class V
0.41––
TUBB2A
tubulin beta 2A class IIa
0.41––
TUBB4B
tubulin beta 4B class IVb
0.41––
TUBB2B
tubulin beta 2B class IIb
0.41––
TUBA1A
tubulin alpha 1a
0.41––
TUBA4A
tubulin alpha 4a
0.41––
TUBA1B
tubulin alpha 1b
0.41––

7 biomarkers in Rhabdomyosarcoma have validated or clinical evidence in CIViC

Accepted evidence from CIViC, the open (CC0) expert-curated knowledgebase, searched by the disease name “Rhabdomyosarcoma”. Level A is validated (for example in guidelines or an approval), level B comes from clinical trials or studies. 38 accepted items exist; the first 38 were read. This is the published evidence, not treatment advice.