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February 21, 2019February 21, 2019

Cannabinoid-Based Treatment for Huntington’s Granted Orphan Drug Designation by FDA

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tMMJ-002, a cannabis investigational product for the treatment of Huntington’s disease, has been granted Orphan Drug designation by the U.S. Food and Drug Administration, announced its developer MMJ International Holdings. This new status ... Read more

February 21, 2019February 20, 2019

Vienna to Host RARE2019 Meeting on Rare Diseases

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About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The ... Read more

February 19, 2019February 19, 2019

Emotional Recognition Defect Linked to Apathy in Huntington’s, Study Shows

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Impairment in emotional recognition — the process of identifying human emotion, most typically from facial expressions, particularly happy ones — seems to be linked with apathy in Huntington’s disease ... Read more

February 14, 2019February 14, 2019

Researchers Get $6 Million to Continue Work on Huntington’s Stem Cell Therapy

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In support of ongoing development of a prospective new therapy for Huntington’s disease, the California Institute for Regenerative Medicine (CIRM) has awarded University of California, Irvine (UCI) investigators $6 ... Read more

February 12, 2019February 12, 2019

Fast Screening Strategy Identifies Potential New Inhibitors for Huntington’s Disease

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Several small molecules have been identified by the Alborada Drug Discovery Institute as potential inhibitors to help halt the underlying mechanisms that drive the development of neurodegenerative diseases such as Huntington’s and Parkinson’s ... Read more

February 11, 2019

Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

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Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run ... Read more

February 7, 2019February 7, 2019

Impaired Glial Cell Development Contributes To Huntington’s, Mouse Study Suggests

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Dysfunction in the development and maturation of the brain’s primary support cells, know as glial cells, contributes to nerve cell damage in a a mouse model of Huntington’s disease. “These new ... Read more

February 5, 2019February 5, 2019

Phase 3 Trial of RG6042 Will Gauge Potential of Gene-silencing Approaches for Huntington’s, Other Diseases

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The upcoming Phase 3 trial of RG6042 will test whether lowering the levels of a mutant form of huntingtin (mHTT) translates into functional, motor and cognitive benefits for people ... Read more

January 31, 2019February 1, 2019

Huntington’s Patients in US and Canada Being Enrolled in Phase 3 Study of RG6042

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A Phase 3 clinical trial is enrolling adults with Huntington’s disease across the United States and Canada to test the potential of the investigational therapy RG6042. The GENERATION HD1 ... Read more

January 29, 2019January 29, 2019

FDA Clears First Clinical Study of AMT-130 Gene Therapy in Huntington’s disease

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uniQure is launching a Phase 1/2 trial in the United States to evaluate the safety and effectiveness of its Huntington’s disease investigational gene therapy AMT-130. This follows recent approval of ... Read more

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