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Please use this identifier to cite or link to this item: http://repository.li.mahidol.ac.th/dspace/handle/123456789/34877
Title: Managing tuberous sclerosis in the Asia-Pacific region: Refining practice and the role of targeted therapy
Authors: John A. Lawson
Chi Fung Chan
Ching Shiang Chi
Pi Chuan Fan
Heung Dong Kim
Ki Joong Kim
Surachai Likasitwatanakul
Marilyn Ortiz
Kate Riney
Stacey Kiat Hong Tay
Chee Kian Tham
University of New South Wales (UNSW) Australia
The University of Hong Kong
Tungs' Taichung MetroHarbor Hospital
National Taiwan University Hospital
Yonsei University College of Medicine
Seoul National University
Mahidol University
Philippine Children's Medical Center
St. Luke's Medical Center Quezon City
Mater Children's Hospital
Yong Loo Lin School of Medicine
National University Health System
National Cancer Centre, Singapore
Keywords: Medicine;Neuroscience
Issue Date: 1-Jan-2014
Citation: Journal of Clinical Neuroscience. Vol.21, No.7 (2014), 1180-1187
Abstract: Tuberous sclerosis complex (TSC) is a multisystem genetic disorder, with heterogeneous manifestations that pose major diagnostic and management challenges and incur considerable chronic disease burden on patients, their caregivers and healthcare systems. This survey of clinical practice in the Asia-Pacific region highlights priorities for improving TSC management in the region. The prevalence of TSC in non-Caucasians is uncertain and more data are needed to assess its impact and health-economic burden. There are unmet needs for access to genetic testing and earlier diagnosis and intervention. TSC management is multidisciplinary and largely based on experience, backed by international guidelines; however, physicians in the Asia-Pacific region feel isolated and lack local or regional guidance and support structures to implement best-practice. Raising awareness of TSC and increasing trans-regional collaboration are particular priorities. Understanding of TSC pathophysiology has enabled the development of targeted therapies. Encouraging data indicate that mammalian target of rapamycin (mTOR) inhibitors can ameliorate TSC-related lesions and may potentially change the treatment paradigm. Ultimately, improving outcomes for TSC patients in the region requires greater collaboration and a holistic, patient-focused, continuum of care that is maintained through the transition from pediatric to adult care. © 2013 Elsevier Ltd. All rights reserved.
URI: https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=84901229579&origin=inward
http://repository.li.mahidol.ac.th/dspace/handle/123456789/34877
ISSN: 15322653
09675868
Appears in Collections:Scopus 2011-2015

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