Optimizing biologically targeted clinical trials for neurofibromatosis
作者： David H Gutmann , Jaishri O Blakeley , Bruce R Korf , Roger J Packer , None
DOI: 10.1517/13543784.2013.772979
关键词: Genetically Engineered Mouse 、 Neurofibromatoses 、 Bioinformatics 、 Neurofibromin 1 、 Clinical study design 、 Pathology 、 Medicine 、 MEDLINE 、 Neurofibromatosis 、 Neurofibromatosis type 2 、 Clinical trial
摘要: Introduction: The neurofibromatoses (neurofibromatosis type 1, NF1 and neurofibromatosis 2, NF2) comprise the most common inherited conditions in which affected children adults develop tumors of central peripheral nervous system. In this review, authors discuss how establishment Neurofibromatosis Clinical Trials Consortium (NFCTC) has positively impacted on design execution treatment studies for individuals with NF2. Areas covered: Using an extensive PUBMED search collaboration select NFCTC members expert distinct NF topics, clinical features NF2, molecular biology NF2 genes, development application clinically relevant Nf1 Nf2 genetically engineered mouse models formation to enable efficient trial execution. Expert opinion: resulted a more seamless integration preclinical human trials efforts. Leve...
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