News

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 and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…

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 brains of nonhuman primates and targeted specific areas involved in cognition and psychiatric function. This study, “…

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 positive preclinical results, together with other ongoing studies, are expected to support the submission of an investigational new…

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, a study shows. The study’s authors think such driving simulators could be useful screening tools to aid clinicians in…

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, “Task-switching abilities in pre-manifest Huntington’s disease subjects” was published in Parkinsonism and Related Disorders. Although family history and DNA…

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 recent research. The study, “Evaluation of mutant huntingtin and neurofilament proteins as potential markers in Huntington’s disease,” was published…

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 Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…

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 human aging and neurodegenerative diseases, including Huntington’s. The five-year grant is one of the NIH’s Program Project Grants, designed to assemble…

Measuring concentrations of mutant huntingtin (mHTT) protein and another protein, called neurofilament light protein (NfL), can provide early and specific indications of Huntington’s-related changes, according to a new study. The findings suggest the potential of these two markers in both clinical practice and clinical research. The study, “…

A Georgetown University Medical Center (GUMC) Phase 1b clinical trial is recruiting patients to test the safety and tolerability of a leukemia treatment called nilotinib in Huntington’s disease patients. The research team plans to initially enroll 10 patients with early to moderate Huntington’s. Patients will receive 150mg of…