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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

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