TY - JOUR
T1 - Advances and future directions for tuberous sclerosis complex research
T2 - Recommendations from the 2015 strategic planning conference
AU - Sahin, Mustafa
AU - Henske, Elizabeth P.
AU - Manning, Brendan D.
AU - Ess, Kevin C.
AU - Bissler, John J.
AU - Klann, Eric
AU - Kwiatkowski, David J.
AU - Roberds, Steven L.
AU - Silva, Alcino J.
AU - Hillaire-Clarke, Coryse St
AU - Young, Lisa R.
AU - Zervas, Mark
AU - Mamounas, Laura A.
N1 - Funding Information:
Funding support for the March 2015 workshop on which this article reports (“Unlocking Treatments for TSC: 2015 Strategic Plan”) was provided by the National Institute of Neurological Disorders and Stroke (NINDS) and the Tuberous Sclerosis Alliance . M.S. is supported by the NIH U01 NS082320 and the Developmental Synaptopathies Consortium ( U54NS092090 ), which is a part of the NCATS Rare Diseases Clinical Research Network (RDCRN). RDCRN is an initiative of the Office of Rare Diseases Research (ORDR), NCATS, funded through collaboration between NCATS, NIMH, NINDS and NICHD. E.P.H. is supported by the Lucy J. Engles Program in TSC and LAM Research. K.C.E. was supported by NIH 1R01 NS078289. Research in the laboratory of B.D.M. related to the subject of this report was supported by NIH grant R35-CA197459 and to B.D.M. and D.J.K by P01-CA120964 . M.Z. was an Amgen employee at the time, the work was performed and fully supported by Amgen in the form of salary. L.R.Y. was supported by an Established Investigator Award from the LAM Foundation and by the Rare Lung Diseases Consortium ( U54HL127672 ), which is part of the Rare Diseases Clinical Research Network (RDCRN), an initiative of the Office of Rare Diseases Research (ORDR), NCATS, funded by collaboration between NCATS and NHLBI. M.S. has received research grants from Novartis . B.D.M. is on the scientific advisory boards for LAM Therapeutics and Navitor Pharmaceuticals, but these roles did not influence the content of this article. M.S. is an Associate Editor for this journal but did not take part in the review and publication decisions.
Publisher Copyright:
© 2016 Elsevier Inc. All rights reserved.
PY - 2016/7/1
Y1 - 2016/7/1
N2 - On March 10 to March 12, 2015, the National Institute of Neurological Disorders and Stroke and the Tuberous Sclerosis Alliance sponsored a workshop in Bethesda, Maryland, to assess progress and new opportunities for research in tuberous sclerosis complex with the goal of updating the 2003 Research Plan for Tuberous Sclerosis (http://www.ninds.nih.gov/about-ninds/plans/tscler-research-plan.htm). In addition to the National Institute of Neurological Disorders and Stroke and Tuberous Sclerosis Alliance, participants in the strategic planning effort and workshop included representatives from six other Institutes of the National Institutes of Health, the Department of Defense Tuberous Sclerosis Complex Research Program, and a broad cross-section of basic scientists and clinicians with expertise in tuberous sclerosis complex along with representatives from the pharmaceutical industry. Here we summarize the outcomes from the extensive premeeting deliberations and final workshop recommendations, including (1) progress in the field since publication of the initial 2003 research plan for tuberous sclerosis complex, (2) the key gaps, needs, and challenges that hinder progress in tuberous sclerosis complex research, and (3) a new set of research priorities along with specific recommendations for addressing the major challenges in each priority area. The new research plan is organized around both short-term and long-term goals with the expectation that progress toward specific objectives can be achieved within a five to ten year time frame.
AB - On March 10 to March 12, 2015, the National Institute of Neurological Disorders and Stroke and the Tuberous Sclerosis Alliance sponsored a workshop in Bethesda, Maryland, to assess progress and new opportunities for research in tuberous sclerosis complex with the goal of updating the 2003 Research Plan for Tuberous Sclerosis (http://www.ninds.nih.gov/about-ninds/plans/tscler-research-plan.htm). In addition to the National Institute of Neurological Disorders and Stroke and Tuberous Sclerosis Alliance, participants in the strategic planning effort and workshop included representatives from six other Institutes of the National Institutes of Health, the Department of Defense Tuberous Sclerosis Complex Research Program, and a broad cross-section of basic scientists and clinicians with expertise in tuberous sclerosis complex along with representatives from the pharmaceutical industry. Here we summarize the outcomes from the extensive premeeting deliberations and final workshop recommendations, including (1) progress in the field since publication of the initial 2003 research plan for tuberous sclerosis complex, (2) the key gaps, needs, and challenges that hinder progress in tuberous sclerosis complex research, and (3) a new set of research priorities along with specific recommendations for addressing the major challenges in each priority area. The new research plan is organized around both short-term and long-term goals with the expectation that progress toward specific objectives can be achieved within a five to ten year time frame.
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U2 - 10.1016/j.pediatrneurol.2016.03.015
DO - 10.1016/j.pediatrneurol.2016.03.015
M3 - Review article
C2 - 27267556
AN - SCOPUS:84971667697
VL - 60
SP - 1
EP - 12
JO - Pediatric Neurology
JF - Pediatric Neurology
SN - 0887-8994
ER -