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Medline ® Abstract for Reference 28

of 'Neurofibromatosis type 1 (NF1): Management and prognosis'

Optimizing biologically targeted clinical trials for neurofibromatosis.
Gutmann DH, Blakeley JO, Korf BR, Packer RJ
Expert Opin Investig Drugs. 2013 Apr;22(4):443-62. Epub 2013 Feb 21.
INTRODUCTION: The neurofibromatoses (neurofibromatosis type 1, NF1 and neurofibromatosis type 2, NF2) comprise the most common inherited conditions in which affected children and adults develop tumors of the central and peripheral nervous system. In this review, the authors discuss how the establishment of the Neurofibromatosis Clinical Trials Consortium (NFCTC) has positively impacted on the design and execution of treatment studies for individuals with NF1 and NF2.
AREAS COVERED: Using an extensive PUBMED search in collaboration with select NFCTC members expert in distinct NF topics, the authors discuss the clinical features of NF1 and NF2, the molecular biology of the NF1 and NF2 genes, the development and application of clinically relevant Nf1 and Nf2 genetically engineered mouse models and the formation of the NFCTC to enable efficient clinical trial design and execution.
EXPERT OPINION: The NFCTC has resulted in a more seamless integration of mouse preclinical and human clinical trials efforts. Leveraging emerging enabling resources, current research is focused on identifying subtypes of tumors in NF1 and NF2 to deliver the most active compounds to the patients most likely to respond to the targeted therapy.
Washington University School of Medicine, Department of Neurology and Washington University Neurofibromatosis Center, 660 South Euclid Avenue, St. Louis, MO 63110, USA. gutmannd@neuro.wustl.edu