Optimizing biologically targeted clinical trials for neurofibromatosis

Topical Rapamycin Therapy to Alleviate the Cutaneous Manifestations of Tuberous Sclerosis Complex

(2013) Mary Kay Koenig et al.   Drugs in research & development

Deterioration of fracture healing in the mouse model of NF1 long bone dysplasia

(2012) T. El Khassawna et al.   BONE

Innate Neural Stem Cell Heterogeneity Determines the Patterning of Glioma Formation in Children

(2012) Da Yong Lee et al.   CANCER CELL

Deconvoluting mTOR biology

(2012) Jason D. Weber et al.   CELL CYCLE

Internal tumor burden in neurofibromatosis Type I patients with large NF1 deletions

(2012) Lan Kluwe et al.   GENES CHROMOSOMES & CANCER

Sustained MEK inhibition abrogates myeloproliferative disease in Nf1 mutant mice

(2012) Tiffany Chang et al.   JOURNAL OF CLINICAL INVESTIGATION

MEK inhibition exhibits efficacy in human and mouse neurofibromatosis tumors

(2012) Walter J. Jessen et al.   JOURNAL OF CLINICAL INVESTIGATION

Assessment of antiangiogenic effect of imatinib mesylate on vestibular schwannoma tumors using in vivo corneal angiogenesis assay

(2012) Ulaş Yener et al.   JOURNAL OF NEUROSURGERY

Imatinib mesylate for plexiform neurofibromas in patients with neurofibromatosis type 1: a phase 2 trial

(2012) Kent A Robertson et al.   LANCET ONCOLOGY

A murine lung cancer co-clinical trial identifies genetic modifiers of therapeutic response

(2012) Zhao Chen et al.   NATURE

Phase II trial of lapatinib in adult and pediatric patients with neurofibromatosis type 2 and progressive vestibular schwannomas

(2012) Matthias A. Karajannis et al.   NEURO-ONCOLOGY

Visual outcomes in children with neurofibromatosis type 1-associated optic pathway glioma following chemotherapy: a multicenter retrospective analysis

(2012) M. J. Fisher et al.   NEURO-ONCOLOGY

Bevacizumab for Progressive Vestibular Schwannoma in Neurofibromatosis Type 2

(2012) Scott R. Plotkin et al.   OTOLOGY & NEUROTOLOGY

Quantitative Assessment of Whole-Body Tumor Burden in Adult Patients with Neurofibromatosis

(2012) Scott R. Plotkin et al.   PLoS One

Primary osteopathy of vertebrae in a neurofibromatosis type 1 murine model

(2011) Wei Zhang et al.   BONE

An Integrated In Vitro and In Vivo High-Throughput Screen Identifies Treatment Leads for Ependymoma

(2011) Jennifer M. Atkinson et al.   CANCER CELL

Perinatal or Adult Nf1 Inactivation Using Tamoxifen-Inducible PlpCre Each Cause Neurofibroma Formation

(2011) D. A. Mayes et al.   CANCER RESEARCH

Susceptible Stages in Schwann Cells for NF1-Associated Plexiform Neurofibroma Development

(2011) L. Q. Le et al.   CANCER RESEARCH

Binary Regulation of Hippo Pathway by Merlin/NF2, Kibra, Lgl, and Melted Specifies and Maintains Postmitotic Neuronal Fate

(2011) David Jukam et al.   DEVELOPMENTAL CELL

Mice lacking Nf1 in osteochondroprogenitor cells display skeletal dysplasia similar to patients with neurofibromatosis type I

(2011) Weixi Wang et al.   HUMAN MOLECULAR GENETICS

Spinal ependymomas in neurofibromatosis Type 2: a retrospective analysis of 55 patients

(2011) Scott R. Plotkin et al.   JOURNAL OF NEUROSURGERY-SPINE

Plexiform Neurofibromas in Children with Neurofibromatosis Type 1: Frequency and Associated Clinical Deficits

(2011) Rosa Nguyen et al.   JOURNAL OF PEDIATRICS

Scram1 is a modifier of spinal cord resistance for astrocytoma on mouse Chr 5

(2011) Jessica Amlin-Van Schaick et al.   MAMMALIAN GENOME

Bevacizumab induces regression of vestibular schwannomas in patients with neurofibromatosis type 2

(2011) V.-F. Mautner et al.   NEURO-ONCOLOGY

A phase 1 study of vinblastine in combination with carboplatin for children with low-grade gliomas: a Children's Oncology Group phase 1 consortium study

(2011) R. I. Jakacki et al.   NEURO-ONCOLOGY

Identification of a progenitor cell of origin capable of generating diverse meningioma histological subtypes

(2011) M Kalamarides et al.   ONCOGENE

Lovastatin as Treatment for Neurocognitive Deficits in Neurofibromatosis Type 1: Phase I Study

(2011) Maria T. Acosta et al.   PEDIATRIC NEUROLOGY

Merlin/NF2 Functions Upstream of the Nuclear E3 Ubiquitin Ligase CRL4DCAF1 to Suppress Oncogenic Gene Expression

(2011) J. Cooper et al.   Science Signaling

The APL Paradigm and the “Co-Clinical Trial” Project

(2011) Caterina Nardella et al.   Cancer Discovery

Birth incidence and prevalence of tumor-prone syndromes: Estimates from a UK family genetic register service

(2010) D.G. Evans et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Anti–Vascular Endothelial Growth Factor Therapies as a Novel Therapeutic Approach to Treating Neurofibromatosis-Related Tumors

(2010) Hon Kit Wong et al.   CANCER RESEARCH

Merlin in organ size control and tumorigenesis: Hippo versus EGFR?

(2010) C. Yi et al.   GENES & DEVELOPMENT

Reduced striatal dopamine underlies the attention system dysfunction in neurofibromatosis-1 mutant mice

(2010) Jacquelyn A. Brown et al.   HUMAN MOLECULAR GENETICS

Defective cAMP Generation Underlies the Sensitivity of CNS Neurons to Neurofibromatosis-1 Heterozygosity

(2010) J. A. Brown et al.   JOURNAL OF NEUROSCIENCE

Skeletal abnormalities in neurofibromatosis type 1: Approaches to therapeutic options

(2009) Florent Elefteriou et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Cell of Origin and Microenvironment Contribution for NF1-Associated Dermal Neurofibromas

(2009) Lu Q. Le et al.   Cell Stem Cell

Phase II Multicenter Trial of Imatinib in 10 Histologic Subtypes of Sarcoma Using a Bayesian Hierarchical Statistical Model

(2009) Rashmi Chugh et al.   JOURNAL OF CLINICAL ONCOLOGY

Phase II Study of Sorafenib in Patients With Metastatic or Recurrent Sarcomas

(2009) Robert G. Maki et al.   JOURNAL OF CLINICAL ONCOLOGY

Suggested response criteria for phase II antitumor drug studies for neurofibromatosis type 2 related vestibular schwannoma

(2009) Scott R. Plotkin et al.   JOURNAL OF NEURO-ONCOLOGY

NF2/Merlin Is a Novel Negative Regulator of mTOR Complex 1, and Activation of mTORC1 Is Associated with Meningioma and Schwannoma Growth

(2009) M. F. James et al.   MOLECULAR AND CELLULAR BIOLOGY

Response and resistance to MEK inhibition in leukaemias initiated by hyperactive Ras

(2009) Jennifer O. Lauchle et al.   NATURE

THREE-DIMENSIONAL VOLUMETRICS FOR TRACKING VESTIBULAR SCHWANNOMA GROWTH IN NEUROFIBROMATOSIS TYPE II

(2009) Gordon J. Harris et al.   NEUROSURGERY

Hearing Improvement after Bevacizumab in Patients with Neurofibromatosis Type 2

(2009) Scott R. Plotkin et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Conditional Mouse Model for Malignant Mesothelioma

(2008) Johan Jongsma et al.   CANCER CELL

Plexiform and Dermal Neurofibromas and Pigmentation Are Caused by Nf1 Loss in Desert Hedgehog-Expressing Cells

(2008) Jianqiang Wu et al.   CANCER CELL

Validation of the p21-Activated Kinases as Targets for Inhibition in Neurofibromatosis Type 2

(2008) C. Yi et al.   CANCER RESEARCH

Preclinical Cancer Therapy in a Mouse Model of Neurofibromatosis-1 Optic Glioma

(2008) B. Hegedus et al.   CANCER RESEARCH

Pten Haploinsufficiency Accelerates Formation of High-Grade Astrocytomas

(2008) C.-H. Kwon et al.   CANCER RESEARCH

Nf1-Dependent Tumors Require a Microenvironment Containing Nf1+/−- and c-kit-Dependent Bone Marrow

(2008) Feng-Chun Yang et al.   CELL

Neurofibromin Regulation of ERK Signaling Modulates GABA Release and Learning

(2008) Yijun Cui et al.   CELL

How does the Schwann cell lineage form tumors in NF1?

(2008) Steven L. Carroll et al.   GLIA

Cerebrovascular dysplasia in neurofibromatosis type 1

(2008) A G Cairns et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Gliomas in Neurofibromatosis Type 1: A Clinicopathologic Study of 100 Patients

(2008) Fausto J. Rodriguez et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

The Neurofibromatosis 2 Protein, Merlin, Regulates Glial Cell Growth in an ErbB2- and Src-Dependent Manner

(2008) S. S. Houshmandi et al.   MOLECULAR AND CELLULAR BIOLOGY

Generalized metabolic bone disease in Neurofibromatosis type I

(2008) Nicola Brunetti-Pierri et al.   MOLECULAR GENETICS AND METABOLISM