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Pediatr Neurol. 2016 Jul;60:1-12. doi: 10.1016/j.pediatrneurol.2016.03.015. Epub 2016 Apr 2.

Advances and Future Directions for Tuberous Sclerosis Complex Research: Recommendations From the 2015 Strategic Planning Conference.

Author information

1
Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts. Electronic address: mustafa.sahin@childrens.harvard.edu.
2
Division of Pulmonary and Critical Care Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts.
3
Department of Genetics and Complex Diseases, Harvard T.H. Chan School of Public Health, Boston, Massachusetts.
4
Vanderbilt Kennedy Center for Research on Human Development, Department of Pediatrics, Vanderbilt University, Nashville, Tennessee.
5
University of Tennessee Health Science Center, Le Bonheur Children's Hospital and St. Jude Children's Research Hospital, Memphis, Tennessee.
6
Center for Neural Science, New York University, New York, New York.
7
Tuberous Sclerosis Alliance, Silver Spring, Maryland.
8
Departments of Neurobiology, Psychiatry and Psychology, Integrative Center for Learning and Memory, Brain Research Institute, University of California at Los Angeles, Los Angeles, California.
9
National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland.
10
Division of Pulmonary Medicine, Department of Pediatrics, Vanderbilt University School of Medicine, Nashville, Tennessee; Division of Allergy, Pulmonary, and Critical Care, Department of Medicine, Vanderbilt University School of Medicine, Nashville, Tennessee.
11
Department of Neuroscience, Amgen Inc, Cambridge, Massachusetts.
12
National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland. Electronic address: mamounas@ninds.nih.gov.
13
Department of Pharmacology, Weill Cornell Medicine, New York, New York.
14
Department of Dermatology, Uniformed Services University of the Health Sciences, Bethesda, Maryland.
15
Pulmonary, Allergy & Critical Care, Department of Medicinem, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
16
Cardiovascular and Pulmonary Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland.
17
Division of Pediatric Nephrology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
18
Institut Necker-Enfants Malades (INEM), Paris; Institut National de la Santé et de la Recherche Médicale (INSERM), Paris; Université Paris Descartes, Sorbonne, Institut Necker-Enfants Malades (INEM), Paris; Université Paris Descartes, Sorbonne, Paris cedex 14, France; U1151, Paris, France; Paris Cité, Paris, France.
19
National Cancer Institute, National Institutes of Health, Rockville, Maryland.
20
Center for Sarcoma and Bone Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
21
Cincinnati Children's Hospital Medical Center/University of Cincinnati College of Medicine, Department of Pediatrics, Division of Neurology, Cincinnati, Ohio.
22
Nemours/AI DuPont Hospital for Children, Wilmington, Delaware.
23
Department of Cell Biology and Neuroscience Rutgers, The State University of New Jersey Nelson Biological Laboratories, Piscataway, New Jersey.
24
Division of Child & Adolescent Psychiatry, University of Cape Town, Rondebosch, South Africa.
25
Department of Human Genetics, Emory University School of Medicine, Atlanta, Georgia.
26
Department of Cell and Developmental Biology, University of Colorado School of Medicine, Aurora, Colorado.
27
Laboratories of Cognitive Neuroscience, Division of Developmental Medicine, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts; Harvard Graduate School of Education, Cambridge, Massachusetts.
28
UAB Department of Neurology and Pediatrics, Birmingham, Alabama.
29
Department of Neurology, Temple University School of Medicine, Philadelphia, Pennsylvania.
30
Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
31
New Medicines, UCB Biopharma SPRL, Braine-l'Alleud, Belgium.
32
Division of Neurology, Department of Pediatrics Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
33
Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts.
34
Division of Pediatric Neurosurgery, NYU Langone Medical Center, New York, New York.
35
Department of Neurology, Washington University School of Medicine, St. Louis, Missouri.
36
Division of Pediatric Neurology, Mattel Children's Hospital, University of California Los Angeles, Los Angeles, California.
37
Genetics Division, Department of Medicine, Brigham and Women's Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts; Ludwig Center at Dana-Farber/Harvard Cancer Center, Boston, Massachusetts.
38
University of North Carolina at Chapel Hill, Lineberger Comprehensive Cancer Center, Chapel Hill, North Carolina.
39
Novartis Institutes for BioMedical Research, Cambridge Massachusetts.
40
Department of Clinical Genetics, Erasmus Medical Center, Rotterdam, The Netherlands.
41
Division of Medical Genetics, Department of Pediatrics, McGovern Medical School, University of Texas Health Science Center at Houston, Houston, Texas.
42
Department of Genetics, Howard Hughes Medical Institute, Harvard Medical School, Boston, Massachusetts.
43
Molecular and Cell Biology Laboratory, The Salk Institute for Biological Studies, La Jolla, California.

Abstract

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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