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HD InsightsHSG Press Release: May 14, 2009

FOR IMMEDIATE RELEASE

Huntington Study Group to Conduct Therapeutic Research Study in Pre-manifest Huntington’s Disease

May 14, 2009 – Physicians at 10 clinical sites are taking part in a nationwide study investigating coenzyme Q10 in gene positive, premanifest Huntington’s disease. This study is known as PREQUEL (Study in PRE-manifest Huntington’s disease of coenzyme Q10 (UbiquinonE) Leading to preventive trials).

Coenzyme Q10 is a nutritional supplement. The objective of the PREQUEL study is to evaluate the safety and tolerability of coenzyme Q10. A secondary objective is to assess the change from baseline to 20 weeks on biomarkers and DNA repair mechanisms in pre-manifest participants treated with coenzyme Q10.

Research participants who are 18 years of age and older and meet all eligibility criteria will be randomly assigned (like the flip of a coin) to receive 600, 1200 or 2400 mg/day of coenzyme Q10 capsules in a blinded fashion. Participants will be seen over 20 weeks. All participants will initiate treatment at 600 mg/day of coenzyme Q10 and then titrate to their assigned dosage over 4 weeks. Following the 4 week titration all participants will be maintained on their assigned study dosage for an additional 16 weeks of follow-up. Researchers at 10 clinical sites in the United States will enroll a total of 90 research participants who have tested positive for the Huntington’s disease (HD) gene expansion and not have features on a physical exam that would suggest a diagnosis of Huntington’s disease (pre-manifest). Each center will enroll approximately 9 participants.

Huntington’s disease (HD) is an inherited disease of the brain that usually begins between the ages of 30 to 50, and includes motor, cognitive and behavioral signs and symptoms. While there are medications to help relieve some of the disease symptoms, there is no known treatment to slow the progression of HD, which affects about 30,000 people in North America.

The HSG is a worldwide, not-for-profit group of physicians and other clinical researchers who are experienced in the care of Huntington’s disease patients and are dedicated to clinical research of Huntington’s disease. The study is sponsored by the National Institute of Neurological Disorders and Stroke (NINDS) and will be conducted under the direction of Principal Investigator Christopher Ross, MD PhD (Johns Hopkins University) and Co-Principal Investigator Kevin Biglan, MD MPH (University of Rochester) and Michael McDermott, PhD (University of Rochester).

Drs. Christopher Ross and Kevin Biglan state, “This will be the first therapeutic research study in pre-manifest HD and will hopefully lead to larger trials designed to delay the onset of HD.”

There is no cost to participate in the study. Participants will be followed for 20 weeks with participation being evaluated monthly. Persons who have tested positive for Huntington’s disease who are interested in participating in this study should visit the Huntington Study Group website at: www.HuntingtonStudyGroup.org.

press release final version 2.25.2009

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Our mission is to promote, disseminate, and facilitate research on Huntington’s disease. To fulfill this mission, we are guided by an outstanding editorial board that includes representatives from three continents, academia, industry, and the HD community.

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