Enrollment is underway in a Phase 2 clinical trial testing Roche’s therapeutic candidate tominersen in people with early manifest Huntington’s disease and those in the prodromal phase of the disease, when hallmark disease symptoms are not yet present. The study, dubbed GENERATION HD2 (NCT05686551), aims…
The European Medicines Agency (EMA) has granted orphan drug status to SAGE-718 as a potential therapy for Huntington’s disease. The experimental therapy is being developed by Sage Therapeutics for treating cognitive impairment associated with Huntington’s and other neurological disorders. Orphan drug status is given to treatments with the potential to improve…
Austedo XR, a new extended-release formulation of Austedo (deutetrabenazine) has been approved by the U.S. Food and Drug Administration (FDA) to treat adults with chorea — involuntary muscle contractions — associated with Huntington’s disease. Austedo XR contains the same active agent as the original therapy, but is taken just once a day, with…
The Huntington’s Disease Society of America (HDSA) is awarding 56 grants totaling more than $2 million to expand its Centers of Excellence network, which is dedicated to providing comprehensive care to people with Huntington’s disease. Dartmouth–Hitchcock Medical Center in New Hampshire joins last year’s total of 55 grant-funded Centers of Excellence. The…
Treatment with antioxidant compounds called fullerenols showed neuroprotective effects in a fruit fly model of Huntington’s disease, particularly at early ages, according to a new study. These findings add to a number of studies highlighting the role of oxidative stress in Huntington’s, and suggest that fullerenols may have…
Spark Therapeutics and Neurochase have joined forces to develop Neurochase’s proprietary delivery technology for gene therapies that target neurodegenerative diseases, such as Huntington’s disease. The technology, called Convection Enhanced Delivery (CED), allows for a controlled delivery of gene and other therapies directly into specific brain regions with the…
In Huntington’s disease, neurodegeneration in a brain region called the striatum differs not only across different types of neurons, but also across distinct striatal compartments, according to a study of brain samples from a Huntington’s patient and mouse models. The striosomes, the striatal compartment involved in regulating mood, was more…
Reduced levels of oxytocin — a natural hormone produced in the brain’s hypothalamus that also works as a chemical messenger — in the cerebrospinal fluid (CSF) were found in Huntington’s disease patients with cognitive and social impairments, a new study shows. The findings support oxytocin levels as a biomarker of impaired…
Neuron-targeted treatment based on zinc-finger DNA-binding proteins — called ZFPs — was found to lower brain levels of mutant huntingtin (mHTT) protein in a Huntington’s disease mouse model. The zinc-finger proteins rescued behavioral deficits and extended survival in these mice, the study demonstrated. ZFPs are among the most abundant…
The U.S. Food and Drug Administration (FDA) has agreed to review Neurocrine Biosciences’ supplemental new drug application (sNDA) for valbenazine to treat chorea caused by Huntington’s disease. “This sNDA filing advances our effort to bring a potential new treatment option to the many thousands of people experiencing chorea associated with…
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