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…
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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 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…
With the support of Houston Astros pitcher Joe Smith and the University of Texas Health Science Center at Houston (UTHealth), about 100 Huntington’s disease patients and their families got to relax for a few hours and take in a ballgame at Minute Maid Park. The tickets for…
Two new clinical studies of Roche’s investigational therapy RG6042 for Huntington’s disease (HD) are planned to start by the end of 2018 and will begin enrolling participants by early 2019. These studies will help researchers to understand Huntington’s progression and the therapeutic effectiveness of RG6042, which may “potentially be the…
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…
Researchers narrowing in on early neuronal functioning stages of development, well before Huntington’s disease symptoms appear, believe they have found a potentially important path to intervening with the diabetes medication metformin. Their research was conducted in a mouse model of the disease. Metformin, an oral medication normally prescribed to patients…
Short huntingtin (HTT) protein fibers precede the formation of larger deposits that accumulate in the brains of those affected by Huntington’s disease, preclinical results reveal. These findings one day may help researchers predict the onset and course of the disease. The study, “mHTT Seeding Activity: A Marker of…
Huntington’s disease begins early, before birth, but symptoms tend to appear much later in life. Now, researchers working with mice have found a way to reverse so-called “pre-Huntington’s symptoms,” including abnormal nerve cell differentiation, with early therapeutic intervention. This could have important implications for future treatment strategies. The study, “Early postnatal…