HD Insights™

Vol. 13 l Winter 2016

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2 HD Insights, Vol. 13 Welcome to the thirteenth edition of HD Insights, timed for release at the beginning of the 11 th Annual CHDI HD Therapeutics Conference. We are pleased to continue our mission to promote, disseminate, and facilitate research in HD, and grateful to our subscribers, our editorial board, and our sponsors for their generous support of the periodical. For our first edition of 2016, HD Insights introduces a new layout and a renewed emphasis on bringing you the latest in preclinical and clinical HD research. We profile Ionis Pharmaceuticals' antisense oligonucleotide IONIS-HTT Rx , currently in a Phase I/IIa trial. We interview the team of scientists and physicians responsible for the drug's development, and explore Ionis' partnership with Roche Pharmaceuticals in an interview with Dr. Scott Schobel. We highlight a selection of highly cited original research articles from 2014, including new insights into the role of astrocytes in HD pathophysiology, a mutation commonly responsible for HD phenocopy syndromes, and the importance of autophagosome abnormalities in HD. Dr. Alpar Lazar describes his work with Dr. Roger Barker, characterizing the emergence of sleep disturbances in prodromal HD patients. Our colleagues at the Journal of Huntington's Disease continue to advance HD research through excellent scholarship. We selected one article from their recent edition and invited the authors to share their work on the new Genetic Modifiers of Motor Onset Age database. Dr. Lise Munsie reprises her Research Round-Up, and joins our editorial team as a scientific content reviewer. Then, for those who were unable to attend HSG 2015, Shoulson Scholar Mr. George McNally summarizes the highlights of that meeting. Finally, we continue to provide an up-to-date status report on HD clinical trials and the development of novel therapies in the pipeline. HD Insights relies on the support of those firms dedicated to advancing HD therapeutics to continue to bring you the latest on HD research. If you would like to become a supporter, or to submit an article, comment, or suggestion, please send us an email at editor@hdinsights.org. For a free electronic subscription, please send an email to subscribe@hdinsights.org. Thank you for your continued support. - - R A Y D O R S E Y , E D I T O R I N C H I E F H D I N S I G H T S T M R A Y D O R S E Y ' S D I S C L O S U R E S : D R . D O R S E Y R E C E I V E S G R A N T S U P P O R T F R O M P R A N A B I O T E C H N O L O G Y , T E VA , A N D T H E H U N T I N G T O N S T U D Y G R O U P . H E I S A C O N S U L T A N T T O T E VA A N D B I O M A R I N . E D I T O R ' S L E T T E R Copyright © Huntington Study Group 2016. All rights reserved. H D I N S I G H T S Teva CNS is committed to continued research and development of its product portfolio and to the development of medicines aimed at meeting the specific needs of the patient communities it serves. Teva's legacy in CNS is grounded in its commitment to ongoing collaboration with academia, medical institutions and patient advocacy groups to find innovative solutions for patients who live with chronic and debilitating diseases. H D I N S I G H T S T M T H A N K S T E V A F O R I T S G E N E R O U S S U P P O R T

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