HD Insights Volume 16

HD Insights: Volume 16

HD Insights Volume 16 (PDF)

February 22nd, 2017 Heather Hare To read a non-PDF version of HD Insights Vol. 16, click here.

2016: Advances and Challenges in HD Therapeutic Development

February 22nd, 2017 Heather Hare By: Meredith A. Achey, BM The year 2016 brought a number of notable developments in HD research and therapeutic development. While an approved, disease-modifying treatment remains elusive, planning and execution of first-in-human trials investigating some of the most promising advances in therapeutic development continued in earnest. Improvements to existing symptomatic therapies moved another step closer
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Brief report: Pfizer Amaryllis trial ends: negative results lead to termination of extension study

February 22nd, 2017 Heather Hare By: Molly J. Elson, BA Pfizer’s Phase 2 trial of PF-02545920, a PDE-10 inhibitor, failed to find the efficacy for which it hoped. In December, Pfizer announced that the therapeutic intervention in its much-anticipated Amaryllis trial produced negative results. The drug failed to show significant improvement in movement, cognition, or behavior and, as a result,
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Highly Cited HD Research

February 22nd, 2017 Heather Hare In this edition of HD Insights, we take a look at impactful HD research articles of 2015. We searched Thomson Reuters’ “Web of Science” service in late 2016 to identify the five most highly cited original research articles reporting on original HD research published in 2015. We invited the corresponding authors to update us on
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Research Round-Up

February 22nd, 2017 Heather Hare By: Lise Munsie, PhD In pharma… The well-tolerated, FDA-approved drug metformin is an antidiabetic drug that mimics caloric restriction. Jin and colleagues tested their hypothesis that it may also counteract pathways affected by the presence of mHTT using the striatal Q111 cell model.1 Under serum starvation conditions, the drug corrected phenotypes such as decreased ATP
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MEET THE COMPANY: uniQure

February 22nd, 2017 Heather Hare VITAL SIGNS NAME: uniQure STOCK SYMBOL: QURE    SHARE PRICE AS OF 2/17/17: $6.31 52-WEEEK RANGE AS OF 2/17/17: $5.25-$16.40 MARKET CAPITALIZATION: $152 million U.S. HEADQUARTERS: Lexington, MA Dr. Pavlina Konstantinova, a research scientist at uniQure, spoke with HD Insights about the company’s program to develop a gene therapy treatment for HD, using micro RNAs
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MEET THE SCIENTIST: Pavlina Konstantinova, MSc, PhD

February 22nd, 2017 Heather Hare VITAL SIGNS NAME: Pavlina Konstantinova, MSc, PhD TITLE: Director of Emerging Technologies and Huntington Disease Program Leader, uniQure EDUCATION: MSc, Biochemistry and Microbiology, Sofia University, Bulgaria; PhD, Bulgaria Academy of Sciences, Sofia, Bulgaria; Post-doctoral research, RNAi-based gene therapy for HIV-1, Academic Medical Center, Amsterdam Netherlands; Post-doctoral research, Viral miRNA function, Duke University, Durham, NC HOBBIES:
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MEET THE COMPOUND: AMT-130

February 22nd, 2017 Heather Hare By: Meredith A. Achey, BM MANUFACTURER: uniQure COMPOUND: Artificial micro RNA targeting human huntingtin, carried by an adeno-associated virus 5 (AAV5) vector (AAV5-miHTT) MECHANISM OF ACTION: Micro RNAs (miRNAs) target messenger RNA (mRNA) transcripts and lead to silencing of the gene by suppressing translation. uniQure’s design also incorporates DNA promoters to enhance transcription of the
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Highlights from the Journal of HD

February 22nd, 2017 Heather Hare Assistive technology for cognition in HD By: Marleen R. van Walsem, MSc, Emilie I. Howe, MSc and Jan C. Frich, MD, PhD Original Article: van Walsem MR, Howe EI, Frich JC, Andelic N. Assistive Technology for Cognition and Health-related Quality of Life in Huntington’s Disease. J Huntingtons Dis. 2016 Oct 1;5(3):261-270. Assistive technology for cognition
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Thank you to Teva Pharmaceuticals

February 22nd, 2017 Heather Hare HD Insights thanks Teva for its ongoing support 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
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Highlights of HSG 2016: Discovering Our Future

February 22nd, 2017 Heather Hare The Huntington Study Group held its 23rd Annual Meeting and 10th Annual Clinical Research Symposium November 2-5, in Nashville, TN. By: Molly J. Elson, BA HSG 2016 brought together world-renowned clinicians, scientists, coordinators, and community members united by a common passion for improving therapies for HD. Together over the course of several days in November,
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Thank you to Raptor

February 22nd, 2017 Heather Hare HD Insights thanks raptor for its generous support.

Editor’s Letter

February 22nd, 2017 Heather Hare Welcome to the 16th edition of HD Insights, timed for release at the beginning of the first Pan-American Parkinson’s Disease and Movement Disorders Congress in Miami, FL. Now in our sixth year, we continue our mission to promote, disseminate, and facilitate HD research with content that is valuable and informative to the global community of
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Job Opportunity: Deputy Editor, HD Insights

February 3rd, 2017 Heather Hare Job description: If you are passionate about HD research and want to reach nearly 3,000 HD researchers and clinicians around the world, consider joining our team as the Deputy Editor of HD Insights. Oversee the creation of 3-4 editions per year of HD Insights. Responsible for soliciting, editing, and coordinating review and layout of each
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