HD Insights: Volume 9

HD Insights: Volume 9

Research Round-Up: Insights of the Year 2013-2014 (In the Clinic)

December 1st, 2014 rtullio In our final issue for 2014, the HD Insights team wanted to recognize the most influential papers in HD research in the 2013–2014 year. Our staff, Editorial Board, and leading HD researchers nominated the eleven papers below in three categories: lab research, clinical research, and imaging and biomarkers. The HD Insights Editorial Board then voted
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Meet the CEO

December 1st, 2014 rtullio VITAL SIGNS NAME: Julie Smith, BS CURRENT POSITION: President and CEO (designate) of Raptor Pharmaceuticals EDUCATION: Bachelor of Science in Biological and Nutritional Science, Cornell University HOBBIES: Spending time with her two little boys, six and eight Julie Smith came to Raptor Pharmaceuticals in 2012 and currently serves as the company’s President and CEO (designate).
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Meet the Founder

December 1st, 2014 rtullio VITAL SIGNS NAME: Ira Shoulson, MD POSITION: Professor of Neurology, Pharmacology and Human Science; Director, Program for Regulatory Science and Medicine (PRSM); Principal Investigator, FDA-Georgetown Center of Excellence in Regulatory Science and Innovation (CERSI), Georgetown University, Washington, DC EDUCATION: MD, Internal Medicine residency (1971–73), Neurology residency (1975–77), Chief Resident in Medicine and Neurology (1977–78), University
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Meet the Compound: RP103 (cysteamine bitartrate delayed-release)

December 1st, 2014 rtullio By: Meredith A. Achey, BM Manufacturer: Raptor Pharmaceuticals Molecular Formula: C6H13NO6S Molecular Weight: 227 g/mol Mechanism of Action: Multiple potential mechanisms. Cysteamine (Figure 1) and its metabolic dimer cystamine (Figure 2) are present in an equilibrium in the body and important in many processes, including as antioxidants, radioprotective radical scavengers, 1 and in metabolic pathways
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Editor’s Letter

December 1st, 2014 rtullio Welcome to the 9th edition of HD Insights. We are grateful to the Huntington Study Group (HSG), our sponsors, and our over 2,500 subscribers for their continuing support of our mission. In this special edition, we feature Dr. Ira Shoulson, founder and leader of the HSG from 1996 to 2014. Dr. Shoulson’s leadership and vision
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Research Round-Up: Insights of the Year 2013-2014 (In the Lab)

December 1st, 2014 rtullio In our final issue for 2014, the HD Insights team wanted to recognize the most influential papers in HD research in the 2013–2014 year. Our staff, Editorial Board, and leading HD researchers nominated the eleven papers below in three categories: lab research, clinical research, and imaging and biomarkers. The HD Insights Editorial Board then voted
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Meet the Company

December 1st, 2014 rtullio         VITAL SIGNS NAME: Raptor Pharmaceuticals HEADQUARTERS: Novato, CA STOCK PRICE AS OF 10/15/2014: $10.19 52 WEEK RANGE: $7.12-$17.72 MARKET CAPITALIZATION: $639 million EMPLOYEES: ~125 APPROVED COMPOUNDS: Cysteamine bitartrate delayed-release capsules (PROCYSBI®), FDA- approved in 2013 for treatment of nephropathic cystinosis in adults and children age 6 and older.

Highlights from the EHDN 2014 Plenary Meeting

December 1st, 2014 rtullio By: Laura E. Clemens and Erik K.H. Jansson The 8th European HD Network Plenary Meeting was held September 19–21 in Barcelona, Spain, and brought together researchers, clinicians, health professionals, support groups and HD family members, to share the latest in HD research and treatment. Research sessions highlighted recent findings in the biology of HD. Drs.
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Research Round-Up: Insights of the Year 2013-2014 (In Imaging and Biomarkers)

December 1st, 2014 rtullio In our final issue for 2014, the HD Insights team wanted to recognize the most influential papers in HD research in the 2013–2014 year. Our staff, Editorial Board, and leading HD researchers nominated the eleven papers below in three categories: lab research, clinical research, and imaging and biomarkers. The HD Insights Editorial Board then voted
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