Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog-Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032.

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Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog-Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032. is …
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scholarly articleQ13442814

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P356DOI10.1200/JCO.2014.60.1591
P8608Fatcat IDrelease_nhcy6kdjtbf5xhf7aluowqi7xq
P3181OpenCitations bibliographic resource ID956127
P932PMC publication ID4534527
P698PubMed publication ID26169613

P50authorTom CurranQ7815488
Arzu Onar-ThomasQ91519746
Amar GajjarQ92888059
Sue C. KasteQ112113580
Michael D PradosQ114338680
Murali ChintagumpalaQ41599004
Giles RobinsonQ55459192
Maryam FouladiQ61303209
Richard GilbertsonQ64746860
Annick DesjardinsQ73536038
Sridharan GururanganQ73536128
Clinton F StewartQ88219529
P2093author name stringDavid W Ellison
Gang Wu
Tong Lin
Stewart Goldman
Sariah J Allen
Michael Rusch
Roger J Packer
James M Boyett
Ibrahim Qaddoumi
Naoko Takebe
Brent A Orr
P2860cites workMolecular subgroups of medulloblastoma: the current consensusQ24615271
Dissecting the genomic complexity underlying medulloblastomaQ24621907
Smoothened mutation confers resistance to a Hedgehog pathway inhibitor in medulloblastoma.Q27851485
Genome sequencing of SHH medulloblastoma predicts genotype-related response to smoothened inhibitionQ27852966
Subgroup-specific structural variation across 1,000 medulloblastoma genomesQ28271811
Genetic elimination of Suppressor of fused reveals an essential repressor function in the mammalian Hedgehog signaling pathwayQ28594509
Medulloblastoma exome sequencing uncovers subtype-specific somatic mutationsQ30040534
Molecular subgroups of medulloblastoma: an international meta-analysis of transcriptome, genetic aberrations, and clinical data of WNT, SHH, Group 3, and Group 4 medulloblastomasQ34167499
Genome sequencing of pediatric medulloblastoma links catastrophic DNA rearrangements with TP53 mutationsQ34248736
The G protein α subunit Gαs is a tumor suppressor in Sonic hedgehog-driven medulloblastomaQ35100720
Inhibiting the hedgehog pathway in patients with the basal-cell nevus syndromeQ35184597
Novel mutations target distinct subgroups of medulloblastoma.Q36143877
Medulloblastoma: clinicopathological correlates of SHH, WNT, and non-SHH/WNT molecular subgroupsQ36460209
Definition of Disease-Risk Stratification Groups in Childhood Medulloblastoma Using Combined Clinical, Pathologic, and Molecular VariablesQ36476670
Medulloblastoma comprises four distinct molecular variantsQ36920479
Subgroup-specific prognostic implications of TP53 mutation in medulloblastomaQ36929844
Long-term outcomes among adult survivors of childhood central nervous system malignancies in the Childhood Cancer Survivor StudyQ37246499
Phase I study of vismodegib in children with recurrent or refractory medulloblastoma: a pediatric brain tumor consortium studyQ37375947
Cytogenetic prognostication within medulloblastoma subgroupsQ37627692
Hedgehog fights back: mechanisms of acquired resistance against Smoothened antagonistsQ37902802
Small molecule inhibition of GDC-0449 refractory smoothened mutants and downstream mechanisms of drug resistanceQ39625099
Genomics identifies medulloblastoma subgroups that are enriched for specific genetic alterationsQ40339027
Treatment of medulloblastoma with hedgehog pathway inhibitor GDC-0449Q42031842
Interfering with resistance to smoothened antagonists by inhibition of the PI3K pathway in medulloblastoma.Q42282551
PTEN loss mitigates the response of medulloblastoma to Hedgehog pathway inhibitionQ42447112
A phase I, multicenter, open-label, first-in-human, dose-escalation study of the oral smoothened inhibitor Sonidegib (LDE225) in patients with advanced solid tumorsQ42454105
Universal poor survival in children with medulloblastoma harboring somatic TP53 mutationsQ43826621
Pharmacokinetic-pharmacodynamic analysis of vismodegib in preclinical models of mutational and ligand-dependent Hedgehog pathway activationQ44485080
Transient inhibition of the Hedgehog pathway in young mice causes permanent defects in bone structureQ46278000
Neurocognitive consequences of risk-adapted therapy for childhood medulloblastomaQ48477520
Suppression of the Shh pathway using a small molecule inhibitor eliminates medulloblastoma in Ptc1(+/-)p53(-/-) mice.Q52087418
Loss of suppressor-of-fused function promotes tumorigenesis.Q53334228
P433issue24
P407language of work or nameEnglishQ1860
P921main subjectbrainQ1073
medulloblastomaQ1333608
P304page(s)2646-54
P577publication date2015-08-20
P1433published inJournal of Clinical OncologyQ400292
P1476titleVismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog-Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032.
P478volume33

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