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Optimizing biologically targeted clinical trials for neurofibromatosis

, MD PhD, , MD, , MD PhD & , MD
Pages 443-462 | Published online: 21 Feb 2013

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Simge Acar, Amy E. Armstrong & Angela C. Hirbe. (2022) Plexiform neurofibroma: shedding light on the investigational agents in clinical trials. Expert Opinion on Investigational Drugs 31:1, pages 31-40.
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Xiaoqin Yang, Kaushal Desai, Neha Agrawal, Kirti Mirchandani, Sagnik Chatterjee, Eric Sarpong & Shuvayu Sen. (2021) Characteristics, treatment patterns, healthcare resource use, and costs among pediatric patients diagnosed with neurofibromatosis type 1 and plexiform neurofibromas: a retrospective database analysis of a medicaid population. Current Medical Research and Opinion 37:9, pages 1555-1561.
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Sabyasachi Mukhopadhyay, Arpita Maitra & Shouvik Choudhury. (2021) Selumetinib: the first ever approved drug for neurofibromatosis-1 related inoperable plexiform neurofibroma. Current Medical Research and Opinion 37:5, pages 789-794.
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Xiaoqin Yang, Kaushal Desai, Neha Agrawal, Kirti Mirchandani, Sagnik Chatterjee, Eric Sarpong & Shuvayu Sen. (2020) Treatment, Resource Use and Costs Among Pediatric Patients with Neurofibromatosis Type 1 and Plexiform Neurofibromas. Pediatric Health, Medicine and Therapeutics 11, pages 421-428.
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James A. Walker & Meena Upadhyaya. (2018) Emerging therapeutic targets for neurofibromatosis type 1. Expert Opinion on Therapeutic Targets 22:5, pages 419-437.
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Diana Bradford, Karlyne M Reilly, Brigitte C Widemann, Abby Sandler & Shivaani Kummar. (2016) Developing therapies for rare tumors: opportunities, challenges and progress. Expert Opinion on Orphan Drugs 4:1, pages 93-103.
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Nicole M Brossier & David H Gutmann. (2015) Improving outcomes for neurofibromatosis 1–associated brain tumors. Expert Review of Anticancer Therapy 15:4, pages 415-423.
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Prabhumallikarjun Patil, Bojana Borislavova Pencheva, Vinayak Mahesh Patil & Jason Fangusaro. (2022) Nervous system (NS) Tumors in Cancer Predisposition Syndromes. Neurotherapeutics 19:6, pages 1752-1771.
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