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November 8, 2018November 8, 2018

Impaired Energy Metabolism Linked to Age of Onset in Huntington’s Patients, Study Shows

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A deficiency in energy metabolism in the cells of patients with Huntington’s disease can partly explain the variation in age of onset among individuals, a study shows. The study, ... Read more

November 1, 2018November 1, 2018

Juvenile Huntington’s More Aggressive, Leads to Early Symptoms, Study Reports

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The juvenile form of Huntington’s disease, which affects patients younger than 20, is particularly aggressive and associated with an early onset of symptoms, a retrospective study has found. Based on ... Read more

October 29, 2018October 29, 2018

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

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With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit ... Read more

October 25, 2018October 25, 2018

DNA-based Therapy Reduces Anxious Behavior in Huntington’s Mouse Model, Study Shows

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Suppressing the expression of the defective huntingtin gene using antisense oligonucleotides lowered anxiety-like behavior in a mouse model of Huntington’s disease, a study reports. These compounds were also efficiently delivered into the ... Read more

October 23, 2018October 23, 2018

Gene Therapy VY-HTT01 Can Protect Brain Cells from Damage Caused by HTT Faulty Gene, Results Show

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One-time delivery of Voyager Therapeutics’ lead candidate gene therapy, VY-HTT01, effectively reduced the levels of the faulty gene responsible for Huntington’s disease in important brain areas of non-human large primates. These ... Read more

October 18, 2018October 18, 2018

Advanced Huntington’s Patients Have Marked Difficulties in Driving, Study Shows

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Patients at advanced stages of Huntington’s disease have marked difficulties in controlling vehicles in a driving simulator compared to patients who are at a pre-symptomatic stage or healthy subjects, ... Read more

October 15, 2018October 15, 2018

Neurological Dysfunction Detectable Before Huntington’s Diagnosis, Study Finds

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Signs of neurological dysfunction can start long before a clinical diagnosis of Huntington’s disease, a study shows. Early cognitive rehabilitation programs might be useful in the pre-manifest stage of the disease. The study, ... Read more

October 11, 2018October 11, 2018

New Blood Test May Track Early Changes in Huntington’s Disease, Study Finds

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Two proteins present in the blood and brains of patients with Huntington’s disease may be used as biomarkers to detect the earliest changes caused by the disease, according to ... Read more

October 9, 2018October 9, 2018

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

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Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based ... Read more

October 9, 2018October 9, 2018

Northwestern University Receives $12.6 Million to Study Protein Balance Mechanisms in Health and Disease

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The National Institute on Aging at the National Institutes of Health (NIH) has granted $12.6 million to Northwestern University to study the role of protein quality control mechanisms in ... Read more

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