Phase I and phase II sonidegib and vismodegib clinical trials for the treatment of paediatric and adult MB patients: a systemic review and meta-analysis

Medulloblastoma

(2019) Paul A. Northcott et al.   Nature Reviews Disease Primers

Novel molecular subgroups for clinical classification and outcome prediction in childhood medulloblastoma: a cohort study

(2017) Edward C Schwalbe et al.   LANCET ONCOLOGY

Phase I study of oral sonidegib (LDE225) in pediatric brain and solid tumors and a phase II study in children and adults with relapsed medulloblastoma

(2017) Mark W Kieran et al.   NEURO-ONCOLOGY

Sonidegib: mechanism of action, pharmacology, and clinical utility for advanced basal cell carcinomas

(2017) Sachin Jain et al.   OncoTargets and Therapy

Losers of Primary Cilia Gain the Benefit of Survival

(2017) Gladiola Goranci-Buzhala et al.   Cancer Discovery

A Transposon Screen Identifies Loss of Primary Cilia as a Mechanism of Resistance to SMO Inhibitors

(2017) Xuesong Zhao et al.   Cancer Discovery

Risk stratification of childhood medulloblastoma in the molecular era: the current consensus

(2016) Vijay Ramaswamy et al.   ACTA NEUROPATHOLOGICA

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

(2015) Giles W. Robinson et al.   JOURNAL OF CLINICAL ONCOLOGY

Genome Sequencing of SHH Medulloblastoma Predicts Genotype-Related Response to Smoothened Inhibition

(2014) Marcel Kool et al.   CANCER CELL

A Phase I, Multicenter, Open-Label, First-in-Human, Dose-Escalation Study of the Oral Smoothened Inhibitor Sonidegib (LDE225) in Patients with Advanced Solid Tumors

(2014) J. Rodon et al.   CLINICAL CANCER RESEARCH

Cytogenetic Prognostication Within Medulloblastoma Subgroups

(2014) David J.H. Shih et al.   JOURNAL OF CLINICAL ONCOLOGY

Phase I Study of Vismodegib in Children with Recurrent or Refractory Medulloblastoma: A Pediatric Brain Tumor Consortium Study

(2013) A. Gajjar et al.   CLINICAL CANCER RESEARCH

Medulloblastoma molecular dissection

(2013) Marc Remke et al.   CURRENT OPINION IN ONCOLOGY

Recurrence patterns across medulloblastoma subgroups: an integrated clinical and molecular analysis

(2013) Vijay Ramaswamy et al.   LANCET ONCOLOGY

Vismodegib

(2012) C. M. Rudin   CLINICAL CANCER RESEARCH

Novel mutations target distinct subgroups of medulloblastoma

(2012) Giles Robinson et al.   NATURE

Pharmacokinetics of Hedgehog Pathway Inhibitor Vismodegib (GDC-0449) in Patients with Locally Advanced or Metastatic Solid Tumors: the Role of Alpha-1-Acid Glycoprotein Binding

(2011) R. A. Graham et al.   CLINICAL CANCER RESEARCH

Phase I Trial of Hedgehog Pathway Inhibitor Vismodegib (GDC-0449) in Patients with Refractory, Locally Advanced or Metastatic Solid Tumors

(2011) P. M. LoRusso et al.   CLINICAL CANCER RESEARCH

Small Molecule Inhibition of GDC-0449 Refractory Smoothened Mutants and Downstream Mechanisms of Drug Resistance

(2010) Gerrit J. P. Dijkgraaf et al.   CANCER RESEARCH

Medulloblastoma Comprises Four Distinct Molecular Variants

(2010) Paul A. Northcott et al.   JOURNAL OF CLINICAL ONCOLOGY

Interfering with Resistance to Smoothened Antagonists by Inhibition of the PI3K Pathway in Medulloblastoma

(2010) S. Buonamici et al.   Science Translational Medicine

Discovery of NVP-LDE225, a Potent and Selective Smoothened Antagonist

(2010) Shifeng Pan et al.   ACS Medicinal Chemistry Letters

GDC-0449—A potent inhibitor of the hedgehog pathway

(2009) Kirk D. Robarge et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Treatment of Medulloblastoma with Hedgehog Pathway Inhibitor GDC-0449

(2009) Charles M. Rudin et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inhibition of the Hedgehog Pathway in Advanced Basal-Cell Carcinoma

(2009) Daniel D. Von Hoff et al.   NEW ENGLAND JOURNAL OF MEDICINE