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Tuesday, April 19<br />
Scientific Platform Sessions<br />
6:30 a.m.–8:30 a.m. Scientific Platform Sessions<br />
S27: Movement Disorders.<br />
Essential Tremor and Tardive<br />
Dyskinesia<br />
6:30 a.m. S27.001<br />
Open-Label Extension of KINECT:<br />
A Phase 2 Study of Valbenazine<br />
(NBI-98854) for Tardive Dyskinesia —<br />
Mohammed Bari, Raj Shiwach, Roland<br />
Jimenez, Scott Siegert, Christopher O'Brien<br />
6:45 a.m. S27.002<br />
Botulinum Toxin Treatment of Essential<br />
Tremor - A Customized Approach —<br />
Rezvan Rostami, Christopher Chow, Diana<br />
Richardson, Bahman Jabbari, Duarte<br />
Machado<br />
7:00 a.m. S27.003<br />
Pathogenic PARK2 Variants in Early-<br />
Onset Essential Tremor Families —<br />
Lorraine Clark, Nora Hernandez, Ruth<br />
Ottman, Elan Louis<br />
7:15 a.m. S27.004<br />
A Multi-Center, Randomized,<br />
Controlled Trial of Unilateral Focused<br />
Ultrasound Thalamotomy for Essential<br />
Tremor —William Ondo, Jeff Elias<br />
7:30 a.m. S27.005<br />
Closing the Loop in Deep Brain<br />
Stimulation: A Responsive Treatment<br />
for Essential Tremor —Enrico Opri,<br />
Jonathan Shute, Rene Molina, Kelly Foote,<br />
Michael Okun, Aysegul Gunduz<br />
7:45 a.m. S27.006<br />
Exploring Essential Tremor: Results<br />
from a Large Online Survey —Phaedra<br />
Dowell, Rajesh Pahwa, Kelly Lyons<br />
8:00 a.m. S27.007<br />
A Phase 2 Study of Valbenazine<br />
(NBI-98854) for Treatment of Tardive<br />
Dyskinesia: KINECT 2 —Stewart Factor,<br />
Robert Hauser, Daniel Mandri, Julio<br />
Castro-Gayol, Roland Jimenez, Scott<br />
Siegert, Christopher O'Brien<br />
8:15 a.m. S27.008<br />
Deutetrabenazine Effect on Total<br />
Motor Score in Patients with<br />
Huntington Disease (First-HD)—Samuel<br />
Frank, David Stamler, Victor Sung, Daniel<br />
Claassen, Sarah Janicki, Clement Loy, Erin<br />
Furr-Stimming, David Oakes, Claudia Testa<br />
S28: Clinical Trials and<br />
Experimental Therapies in<br />
Neuromuscular Diseases<br />
6:30 a.m. S28.001<br />
Eteplirsen, a Phosphorodiamidate<br />
Morpholino Oligomer (PMO) for<br />
Duchenne Muscular Dystrophy (DMD):<br />
Clinical Update and Longitudinal<br />
Comparison to External Controls on<br />
Six-Minute Walk Test (6MWT) —J.<br />
Mendell, Nathalie Goemans, Louise<br />
Rodino-Klapac, Zarife Sahenk, Linda<br />
Lowes, Lindsay Alfano, K Berry, E Peterson,<br />
S Lewis, Kim Shontz, J Shao, P Duda, C<br />
Donoghue, F Schnell, J Dworzak, Bruce<br />
Wentworth, E. Kaye, E Mercuri, DMD<br />
Italian Network<br />
6:45 a.m. S28.002<br />
Results of North Star Ambulatory<br />
Assessments (NSAA) in the Phase 3<br />
Ataluren Confirmatory Trial in Patients<br />
with Nonsense Mutation Duchenne<br />
Muscular Dystrophy (ACT DMD) —<br />
Katharine Bushby, Jan Kirschner, Xiaohui<br />
Luo, Gary Elfring, Hans Kroger, Peter<br />
Riebling, Tuyen Ong, Robert Spiegel, Stuart<br />
Peltz, Francesco Muntoni<br />
7:00 a.m. S28.003<br />
Thymectomy in Non-thymomatous<br />
Myasthenia Gravis: Results from<br />
MGTX, a Randomized, Controlled<br />
Trial —Gary Cutter, Henry Kaminski, Gil<br />
Wolfe, Immaculada Aban, On behalf of the<br />
MGTX Trial Research Group<br />
7:15 a.m. S28.004<br />
Intravenous Trehalose Improves<br />
Dysphagia and Muscle Function in<br />
Oculopharyngeal Muscular Dystrophy<br />
(OPMD): Preliminary Results of 24<br />
Weeks Open Label Phase 2 Trial —<br />
Zohar Argov, Irit Gliko-Kabir, Bernard Brais,<br />
Yoseph Caraco, Dalia Megiddo<br />
7:30 a.m. S28.005<br />
Genome Therapy of Myotonic<br />
Dystrophy Type 1 IPS Cells for<br />
Development of Autologous Stem Cell<br />
Therapy —Guangbin Xia, Yuanzheng Gao,<br />
Xiuming Guo, Katherine Santostefano,<br />
Karen McFarland, Naohiro Terada, Tetsuo<br />
Ashizawa<br />
7:45 a.m. S28.006<br />
Effect of Deflazacort and Prednisone<br />
Versus Placebo on Muscle Strength<br />
in Boys with Duchenne Muscular<br />
Dystrophy Who Have Lost Ambulation:<br />
Results from the Deflazacort Clinical<br />
Trial Program —Timothy Cunniff, Stephen<br />
Wanaski, Jordan Dubow, James Meyer<br />
8:00 a.m. S28.007<br />
Antiiflammatory and Neuroprotective<br />
Role of Fingolimod in Experimental<br />
Autoimmune Neuritis in Lewis Rats —<br />
Bjoern Ambrosius, Kalliopi Pitarokoili, Lisa<br />
Schrewe, Ralf Gold<br />
8:15 a.m. S28.008<br />
Electrical Impedance Myography and<br />
Quantitative Ultrasound Detect Steroid<br />
Related Improvements in Duchenne<br />
Muscular Dystrophy —Seward Rutkove,<br />
Craig Zaidman, Jim Wu, Amy Pasternak,<br />
Lavanya Madabusi, Heather Szelag, Tim<br />
Harrington, Adam Pacheck, Sung Yim, Kush<br />
Kapur, Basil Darras<br />
S29: Pediatric Multiple<br />
Sclerosis<br />
6:30 a.m. S29.001<br />
First Genome-Wide Analysis<br />
in Pediatric Multiple Sclerosis<br />
(MS) Confirms a Role for Adult<br />
MS Risk Variants and Reveals<br />
New Candidates —Lisa Barcellos,<br />
Xiaorong Shao, Brooke Rhead, Milena<br />
Gianfrancesco, Jennifer Graves, Amy<br />
Waldman, Timothy Lotze, Teri Schreiner,<br />
Anita Belman, Benjamin Greenberg, Bianca<br />
Weinstock-Guttman, Gregory Aaen, Jan-<br />
Mendelt Tillema, Janace Hart, Jayne Ness,<br />
Yolanda Harris, Jennifer Rubin, Meghan<br />
Candee, Lauren Krupp, Mark Gorman,<br />
Leslie Benson, Moses Rodriguez, Tanuja<br />
Chitnis, Soe Mar, Adeline Vanderver, Ilana<br />
Kahn, John Rose, Shelly Roalstad, Charlie<br />
Casper, Ling Shen, Hong Quach, Catherine<br />
Metayer, Cathy Schaefer, Emmanuelle<br />
Waubant<br />
6:45 a.m. S29.002<br />
Pediatric Radiologically Isolated<br />
Syndrome: Clinical and Radiological<br />
Outcomes —Naila Makhani, Christine<br />
Lebrun Frenay, Aksel Siva, Clarisse<br />
Carra-Dalliere, Jerome De Seze, Francoise<br />
Durand Dubief, Orhun Kantarci, Megan<br />
Langille, Jean Pelletier, Juan Ignacio<br />
Rojas, Teri Schreiner, Mar Tintore, Ugur<br />
Uygunoglu, Darin Okuda, Daniel Pelletier<br />
7:00 a.m. S29.003<br />
Effect of Obesity and Puberty on Risk<br />
and Age at Onset of Pediatric MS —<br />
Tanuja Chitnis, Jennifer Graves, Bianca<br />
Weinstock-Guttman, Anita Belman, Cody<br />
Olsen, Madhusmita Misra, Gregory Aaen,<br />
Leslie Benson, Meghan Candee, Mark<br />
Gorman, Benjamin Greenberg, Timothy<br />
Lotze, Jayne Ness, Moses Rodriguez,<br />
John Rose, Jennifer Rubin, Teri Schreiner,<br />
Jan-Mendelt Tillema, Charlie Casper,<br />
Emmanuelle Waubant<br />
7:15 a.m. S29.004<br />
Demographic and Clinical<br />
Characteristics of 490 Children and<br />
Adolescents with Multiple Sclerosis<br />
(MS): US Network of Pediatric MS<br />
Centers’ Experience —Anita Belman,<br />
Lauren Krupp, Cody Olsen, John Rose,<br />
Gregory Aaen, Leslie Benson, Tanuja<br />
Chitnis, Mark Gorman, Jennifer Graves,<br />
Yolanda Harris, Timothy Lotze, Jayne Ness,<br />
Shelly Roalstad, Moses Rodriguez, Jan-<br />
Mendelt Tillema, Emmanuelle Waubant,<br />
Bianca Weinstock-Guttman, Charlie Casper<br />
7:30 a.m. S29.005<br />
Maternal Illness in Pregnancy and<br />
Perinatal Exposure to Pesticides Are<br />
Associated with Risk for Pediatric<br />
Onset MS —Jennifer Graves, Tanuja<br />
Chitnis, Bianca Weinstock-Guttman,<br />
Jennifer Rubin, Aaron Zelikovitch, Bardia<br />
Nourbakhsh, Timothy Simmons, Charlie<br />
Casper, Emmanuelle Waubant<br />
7:45 a.m. S29.006<br />
Dietary Factors and Risk of Pediatric<br />
Multiple Sclerosis —Julia Pakpoor,<br />
Brandon Seminatore, Jennifer Graves, Teri<br />
Schreiner, Amy Waldman, Timothy Lotze,<br />
Anita Belman, Benjamin Greenberg, Bianca<br />
Weinstock-Guttman, Gregory Aaen, Jan-<br />
Mendelt Tillema, Janace Hart, Jayne Ness,<br />
Yvonne Harris, Jennifer Rubin, Meghan<br />
Candee, Lauren Krupp, Mark Gorman,<br />
Leslie Benson, Moses Rodriguez, Tanuja<br />
Chitnis, Soe Mar, Ilana Kahn, John Rose,<br />
Shelly Roalstad, Michael Waltz, Charlie<br />
Casper, Emmanuelle Waubant<br />
8:00 a.m. S29.007<br />
The Causal Effect of Serum Vitamin D<br />
Levels on Pediatric Multiple Sclerosis:<br />
A Mendelian Randomization Study —<br />
Brooke Rhead, Milena Gianfrancesco,<br />
Xiaorong Shao, Jennifer Graves, Amy<br />
Waldman, Timothy Lotze, Teri Schreiner,<br />
Anita Belman, Benjamin Greenberg, Bianca<br />
Weinstock-Guttman, Gregory Aaen, Jan-<br />
Mendelt Tillema, Janace Hart, Jayne Ness,<br />
Yolanda Harris, Jennifer Rubin, Meghan<br />
Candee, Lauren Krupp, Mark Gorman,<br />
Leslie Benson, Moses Rodriguez, Tanuja<br />
Chitnis, Soe Mar, Ilana Kahn, John Rose,<br />
Shelly Roalstad, Charlie Casper, Ling Shen,<br />
Hong Quach, Catherine Metayer, Cathy<br />
Schaefer, Lisa Barcellos, Emmanuelle<br />
Waubant<br />
8:15 a.m. S29.008<br />
Associations Between Immune<br />
Markers and Gut Microbiota in<br />
Pediatric Multiple Sclerosis and<br />
Controls —Helen Tremlett, Douglas<br />
Fadrosh, Ali Farqui, Janace Hart, Shelly<br />
Roalstad, Jennifer Graves, Collin Spencer,<br />
Susan Lynch, Scott Zamvil, Emmanuelle<br />
Waubant<br />
90 2016 AAN Annual Meeting Abstract Listing