Accepted for Publication: May 10, 2013.
Published Online: September 2, 2013. doi:10.1001/jamaneurol.2013.3487.
Acquisition of data: Marder.
Analysis and interpretation of data: Marder, Eberly, Tanner, Scarmeas, Oakes, Shoulson.
Drafting of the manuscript: Marder, Oakes.
Critical revision of the manuscript for important intellectual content: Gu, Eberly, Tanner, Scarmeas, Shoulson.
Statistical analysis: Gu, Eberly, Oakes.
Obtained funding: Shoulson.
Administrative, technical, or material support: Shoulson.
Study supervision: Scarmeas.
Conflict of Interest Disclosures: Dr Marder served on the editorial board of Neurology; receives research support from grants NS036630, 1UL1 RR024156-01, PO412196-G, and PO412196-G from the National Institutes of Health (NIH). She received compensation for participating on the steering committee for U01NS052592 and from the Parkinson Disease Foundation, Huntington's Disease Society of America, the Parkinson Study Group, CHDI Foundation Inc, and the Michael J. Fox Foundation. Ms Eberly has received research support from NIH, Department of Defense, Michael J. Fox Foundation, Parkinson Disease Foundation, Cephalon, and Lundbeck. Dr Tanner served on the Scientific Advisory Boards of the Michael J. Fox Foundation and the National Spasmodic Dystonia Association; has received support from Agency for Healthcare and Research Quality, the Brin Foundation, National Institute of Environmental Health Sciences, National Institute of Neurological Disorders and Stroke (NINDS), Parkinson’s Disease Foundation, Parkinson’s Institute and Clinical Center, and US Army Medical Research Acquisition Activity (Telemedicine & Advanced Technology Research Center managed through the Neurotoxin Exposure Treatment Research program); is a consultant for Adamas Pharmaceuticals with money to Parkinson’s Institute and Clinical Center and Impax Pharmaceuticals. Dr Oakes has received research support from NINDS, the Department of Defense, and the Michael J. Fox Foundation for studies in Parkinson disease and Huntington disease; served as a consultant for Novo Nordisk Inc on an unrelated project. Dr Shoulson has consulting and advisory board membership with honoraria from the following: Alkermes Inc, Auspex Pharmaceuticals, AZTherapies, Biogen Idec, Clarion Healthcare Consulting LLC, Corporate Meeting Solutions for Update in Neurology Conference, Edison Pharmaceuticals, Envoy, Impax, Ipsen, JAMA Neurology, Johns Hopkins University, Johnson & Johnson, Knopp Biosciences LLC, Lundbeck, Medtronic, Michael J. Fox Foundation, Omeros Corporation, Partners Health Care, Prana Biotechnology, Salamandra, Seneb Biosciences Inc, Shire HGT Inc, University of California, Irvine; receives grants (to Georgetown University and the University of Rochester)from the Food and Drug Administration, The Johns Hopkins University, National Institutes of Health (National Human Genome Research Institute [(NHGRI] and NINDS), and the Parkinson Disease Foundation. Neither Dr Shoulson nor his immediate family received any personal remuneration from any of the sponsors that provide grant support to the University of Rochester, New York, New York, or Georgetown University, Washington, DC. US Government Sponsors are the National Institutes of Health (2 R01 HG002449-06), including support from the NHGRI and the NINDS or from Cure Huntington Disease Initiative, Huntington’s Disease Society of America, Hereditary Disease Foundation, Huntington Society of Canada, and the Fox Family Foundation.
Group Information: The Huntington Study Group PHAROS Investigators are Steering Committee: Ira Shoulson, MD (principal investigator), Karl Kieburtz, MD, MPH (director, Clinical Trials Coordination Center), David Oakes (chief biostatistician), Elise Kayson, MS, RNC (project coordinator), Hongwei Zhao, ScD, M. Aileen Shinaman, JD (Huntington Study Group executive director), Megan Romer, MS, University of Rochester, Rochester, New York; Anne Young, MD, PhD (co-principal investigator), Steven Hersch, MD, PhD, Jack Penney, MD (deceased), Massachusetts General Hospital, Charlestown; Kevin Biglan, MD (medical monitor), The Johns Hopkins University, Baltimore, Maryland; Karen Marder, MD, MPH, Columbia University Medical Center, New York, New York; Jane Paulsen, PhD, University of Iowa, Iowa City; Kimberly Quaid, PhD, Indiana University School of Medicine, Indianapolis; Eric Siemers, MD, Lilly Corporate Center, Indianapolis, Indiana; Caroline Tanner, MD, The Parkinson’s Institute, Sunnyvale, California. Participating Investigators and Coordinators: William Mallonee, MD, David Palmer, MD (deceased), Greg Suter, BA, Hereditary Neurological Disease Centre, Wichita, Kansas; Richard Dubinsky, MD, Gary Gronseth, MD, R. Neil Schimke, MD, Carolyn Gray, RN, University of Kansas Medical Center, Kansas City; Martha Nance, MD, Scott Bundlie, MD, Dawn Radtke, RN, Hennepin County Medical Center/Minneapolis, Minnesota; Sandra Kostyk, MD, PhD, George W. Paulson, MD, Karen Thomas, DO, Nonna Stepanov, MD, Corrine Baic, BS, Ohio State University, Columbus; James Caress, MD, Francis Walker, MD, Vicki Hunt, RN, Wake Forest University School of Medicine, Winston-Salem, North Carolina; Sylvain Chouinard, MD, Guy Rouleau, MD, PhD, Hubert Poiffaut, RN, Brigitte Rioux (deceased), Hotel-Dieu Hospital-Centre hospitalier de l’universite de Montreal, Montreal, Quebec, Canada; Claudia Testa, MD, PhD, Timothy Greenamyre, MD, PhD, Joan Harrison, RN, Emory University School of Medicine, Atlanta, Georgia; Jody Corey-Bloom, MD, PhD, David Song, MD, Guerry Peavy, PhD, Jody Goldstein, BS, University of California, San Diego, LaJolla; Jane Paulsen, PhD, Henry Paulson, MD, Robert L. Rodnitzky, MD, Ania Mikos, BA, Becky Reese, BS, Laura Stierman, BS, Katie Williams, BA, Lynn Vining, RN, MSN, University of Iowa; Karen Marder, MD, MPH, Elan Louis, MD, MSc, Carol Moskowitz, RN, Columbia University Medical Center; Kimberly Quaid, PhD, Joanne Wojcieszek, MD, Melissa Wesson, MS, Indiana University School of Medicine; Ali Samii, MD, Thomas Bird, MD, Hillary Lipe, ARNP, University of Washington & VA Puget Sound Health Care System, Seattle; Norman Reynolds, MD, Karen Blindauer, MD, Jeannine Petit, ANP, Medical College of Wisconsin, Milwaukee; Peter Como, PhD, Frederick Marshall, MD, Timothy Counihan, MD, Kevin Biglan, MD, Carol Zimmerman, RN, University of Rochester; Penelope Hogarth, MD, John Nutt, MD, Pamela Andrews, BS, CCRC, Oregon Health & Science University, Portland; Steven Hersch, MD, PhD, Leslie Shinobu, MD, PhD, Diana Rosas, MD, Yoshio Kaneko, BA, Sona Gevorkian, MS, Paula Sexton, BA, CCRA, Massachusetts General Hospital; John Caviness, MD, Charles Adler, MD, PhD, Mayo Clinic Scottsdale, Scottsdale, Arizona; Vicki Wheelock, MD, David Richman, MD, Teresa Tempkin, RNC, MSN, University of California Davis, Sacramento; Chuang-Kuo Wu, MD, PhD, Hubert Fernandez, MD, Joseph H. Friedman, MD, Margaret Lannon, RN, MS, Brown University (Memorial Hospital of Rhode Island), Pawtucket; Lauren Seeberger, MD, Christopher O’Brien, MD, Sherrie Montellano, MA, Colorado Neurological Institute, Englewood; Ninith Kartha, MD, Sharin Sakurai, MD, PhD, Susan Hickenbottom, MD, PhD, Roger Albin, MD, PhD, Kristine Wernette, RN, MS, University of Michigan, Ann Arbor; Brad Racette, MD, Joel S. Perlmutter, MD, Laura Good, BA, Washington University, St Louis, Missouri; George Jackson, MD, PhD, Susan Perlman, MD, Shelley Segal, MD, Russell Carroll, MA, Laurie Carr, BS, UCLA Medical Center, Los Angeles, California; Wayne Martin, MD, Ted Roberts, MD, Marguerite Wieler, BSC, PT, University of Alberta, Edmonton, Alberta, Canada; Blair Leavitt, MD, Lorne Clarke, MD, CM, Lynn Raymond, MD, PhD, Joji Decolongon, MSC, Vesna Popovska, MD, Elisabeth Almqvist, RN, PhD, University of British Columbia, Vancouver, British Columbia, Canada; William Ondo, MD, Madhavi Thomas, MD, Tetsuo Ashizawa, MD, Joseph Jankovic, MD, Baylor College of Medicine, Houston, Texas; Robert Hauser, MD, Juan Sanchez-Ramos, MD, PhD, Karen Price, MA, Holly Delgado, RN, University of South Florida, Tampa; Sarah Furtado, MD, PhD, Anne Louise LaFontaine, MD, Oksana Suchowersky, MD, Mary Lou Klimek, RN, MA, University of Calgary, Calgary, Alberta, Canada; Rustom Sethna, MD, Mark Guttman, MD, Sandra Russell, BSW, RSW, Sheryl Elliott, RN, Centre for Addiction and Mental Health, Markham, Ontario, Canada; Marc Mentis, MB, CHB, Andrew Feigin, MD, Marie Cox, RN, BSN, Barbara Shannon, RN, North Shore University Hospital, Manhasset, New York; Alan Percy, MD, Leon Dure, MD, Donna Pendley, RN, Jane Lane, RN, BSN, University of Alabama at Birmingham; Madaline Harrison, MD, Elke Rost-Ruffner, RN, BSN, University of Virginia, Charlottesville; William Johnson, MD, University of Medicine and Dentistry of New Jersey Robert Wood Johnson Medical Center, Stratford; Amy Colcher, MD, Andrew Siderowf, MD, Mary Matthews, RN, University of Pennsylvania, Philadelphia; Danna Jennings, MD, Kenneth Marek, MD, Karen Caplan, MSW, Institute for Neurodegenerative Disorders, New Haven, Connecticut; Stewart Factor, DO, Donald Higgins, MD, Eric Molho, MD, Constance Nickerson, LPN, Sharon Evans, LPN, Diane Brown, RN (deceased), Albany Medical College, Albany, New York; Douglas Hobson, MD, Paul Shelton, MD, Shaun Hobson, RN, Winnipeg Clinic, Winnipeg, Manitoba, Canada; Carlos Singer, MD, Nestor Galvez-Jimenez, MD, William Koller, MD (deceased), Doris Martin, DDS, Kelly Lyons, PhD, Dinorah Rodriguez, RN, University of Miami, Miami, Florida; Kathleen Shannon, MD, Cynthia Comella, MD, Jean Jaglin, RN, CCRC, Rush Presbyterian–St Luke’s Medical Center, Chicago, Illinois; Karen Anderson, MD, William Weiner, MD (deceased), Kelly Dustin, RN, BSN, University of Maryland School of Medicine; Adam Rosenblatt, MD, Christopher Ross, MD, PhD, Deborah Pollard, The Johns Hopkins University; Marie H. Saint-Hilaire, MD, Peter Novak, MD, J. Stephen Fink, MD, PhD (deceased), Bonnie Hersh, MD, Melissa Diggin, MS, RN, Leslie Vickers, RN, MS, Boston University, Botson; Wallace Deckel, PhD, James Duffy, MD, Mary Jane Fitzpatrick, APRN, University of Connecticut, Hartford. Participating NIH Authors: Elizabeth Thomson, PhD, National Human Genome Research Institute, Bethesda, Maryland; National Institute of Neurological Disorders and Stroke, Bethesda. Event Monitoring Committee: Steven Hersch, MD, PhD (cochair), Massachusetts General Hospital; Julie Stout, PhD (co-chair), James Calhoun, Indiana University; William Coryell, MD, Cheryl Erwin, JD, PhD, University of Iowa; Vicki Hunt, RN, Wake Forest University School of Medicine; Christopher Ross, MD, PhD, The Johns Hopkins University; Dorothy Vawter, PhD, Minnesota Center for Health Care Ethics. Ethics Committee: Lori Andrews, JD, Debbie Bury, James Calhoun, Chicago-Kent College of Law, Chicago, Illinois; Steven Hersch, MD, PhD (chair), Massachusetts General Hospital; Vicki Hunt, RN, Carl Leventhal, MD, Wake Forest University School of Medicine; Kimberly Quaid, PhD, Indiana University School of Medicine; Aileen Shinaman, JD, University of Rochester; Dorothy Vawter, PhD, Minnesota Center for Health Care Ethics; Nancy Wexler, PhD, Columbia University, New York. Biostatistics and Clinical Trials Coordination Center: Alicia Brocht, BA, Susan Daigneault, Karen Gerwitz, BS, Connie Orme, BA, Ruth Nobel, Victoria Ross, MA, Mary Slough, Arthur Watts, BS, Joe Weber, BS, Christine Weaver, Elaine Julian-Baros, University of Rochester. Genetic/ Environmental Modifiers Committee: Anne Young, MD, PhD (chair), Massachusetts General Hospital; Karen Marder, MD (co-chair), Columbia University Medical Center; Tatiana Foroud, PhD, Indiana University School of Medicine; James Gusella, PhD, Massachusetts General Hospital; David Housman, PhD, Massachusetts Institute of Technology, Boston; Marcy MacDonald, PhD, Massachusetts General Hospital; Richard Myers, PhD, Boston University; Caroline Tanner, MD, The Parkinson’s Institute; Rudolph Tanzi, PhD, Massachusetts General Hospital. Independent Monitoring Committee: Stanley Fahn, MD, Columbia University; Michael Conneally, PhD (deceased), Indiana University; Weiu-Yann Tsai, PhD, Columbia University. Scientific Advisory Committee: Flint Beal, MD, New York Hospital Department of Neurology, New York; David Housman, PhD, Massachusetts Institute of Technology, Boston; Christopher Ross, MD, PhD, The Johns Hopkins University; Rudolph Tanzi, PhD, Anne Young, MD, PhD, Massachusetts General Hospital; Claudia Kawas, MD, University of California, Irvine; Marie Francoise-Chesselet, MD, PhD, University of California Los Angeles. DNA Oversight Committee: Michael Conneally, PhD (deceased), Indiana University Medical Center; Martha Nance, MD, University of Minnesota/Minnesota VA Medical Center; Clifford Shults, MD (deceased), University of California, San Diego; Caroline Tanner, MD, The Parkinson’s Institute. Independent Rater Video Committee: Penelope Hogarth, MD, Oregon Health & Science University; Diana Rosas, MD, Massachusetts General Hospital; Hongwei Zhao, ScD, University of Rochester.
Additional Contributions: A special thanks to all the PHAROS coordinators, without whom this effort would have been impossible. We also thank all the participants for their tremendous dedication to this project.
Correction: This article was corrected online September 6, 2013, for the inappropriate insertion of an article (the) in the Importance section of the Abstract.