Editor’s Letter

Welcome to the 11th edition of HD Insights. We are pleased to continue our mission to promote, disseminate, and facilitate research in Huntington disease. We are grateful to the Huntington Study Group (HSG), our sponsors, and our more than 2800 subscribers for their continuing support.

This issue investigates potential therapeutic strategies for HD treatment and provides a detailed update on disease-modifying strategies set to enter clinical trials in the upcoming year. We discuss Teva’s recent acquisition of Auspex following the promising results of FIRST-HD released earlier this year. We bring you an interview with former FDA attorney Mr. Frank Sasinowski, on his work with the 1983 Orphan Drug Act, his hopes for orphan drug development, and his advice for researchers and companies working to develop new therapies for rare diseases. Dr. Shaun Sanders describes the therapeutic potential of increasing palmitoylation of mHTT, thereby decreasing its toxicity and aggregation within HD patients. Dr. Dagmar Ehrnhoefer details several studies showing that wild-type HTT plays an important role in myristoylation and autophagy, and promotes the clearance of misfolded mHTT. Dr. Lise Munsie summarizes ongoing HD research and Dr. Daniel Zielonka discusses promising diseasemodifying strategies aimed at lowering HTT. Dr. Francesca Cicchetti shares the highlights of February’s CHDI meeting. Finally, we continue to provide an up-to-date status report on ongoing and recently concluded HD clinical trials.

The HD Insights team continues to rely on the support of firms dedicated to developing novel treatments to support our efforts to reach and educate the international HD research community. If you are interested in becoming a supporter, please contact me at editor@hdinsights.org. We welcome new contributors, suggestions for topics, and ideas about how we can better serve you. If you or someone you know would like to share their ideas with over 2800 HD researchers and clinicians around the world, please contact me at editor@hdinsights.org . Finally, subscription to HD Insights is always free: simply send us an email at subscribe@hdinsights.org.

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Ray Dorsey, MD

Editor, HD INSIGHTSTM

HD DISCLOSURES: DR. DORSEY RECEIVES GRANT SUPPORT FROM AUSPEX PHARMACEUTICALS, THE HUNTINGTON STUDY GROUP, AND PRANA BIOTECHNOLOGY.


 

Editorial Board Members

Ray Dorsey, MD — Editor, University of Rochester

Donald Kennedy, PhD — Stanford University

Nobuyuki Nukina, MD, PhD — Riken Brain Science Institute

Rodrigo Osorio — Agrupación Chilena de Huntington

Bernard Ravina, MD — Voyager Therapeutics

Ralf Reilmann, MD, PhD — George Huntington Institute

Sarah Tabrizi, MBChB, PhD — University College London

Leslie Thompson, PhD — University of California Irvine

Huntington Study Group

Shari Kinel, JD — HSG Executive Director

Liz McCarthy, BA — HSG Director of Programs & Communications

Kristin Strazdins — HSG Accounting and Office Manager

Contributors

Francesca Cicchetti, PhD

Dagmar E. Ehrnhoefer, PhD

Ryan E. Korn, BA

Lise Munsie, PhD

Shaun S. Sanders, PhD

Daniel C. Zielonka, MD, PhD

Publication Staff

Meredith A. Achey, BM — Deputy Editor

Ryan E. Korn, BA — Associate Editor

Robin Taylor — Production Editor

Martin Holmes — Technical Editor

David Kolko — Distribution Specialist