A Genetic Lottery
— Rebecca Field

Using machine learning, a form of artificial intelligence, researchers were able to cluster people with Huntington’s disease into three groups based on the speed at which the disease progresses, a study showed. “Machine learning approaches such as this…

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…

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…

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 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…

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…

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.

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…

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…

Researchers have developed a new therapy that reduced the toxic buildup of the mutant huntingtin (HTT) protein — the hallmark of Huntington’s disease — in lab-grown neurons from Huntington’s patients and in a mouse model of the disease. The…

Branaplam (LMI070), an experimental oral therapy for Huntington’s disease that’s currently in early clinical trials, reduces levels of the mutant huntingtin protein by interfering with a process called mRNA splicing, a new study reveals. “Here, we delineated the…

The pharmaceutical company Teva is turning to social media to help people with Huntington’s disease (HD) to improve the management of chorea — a hallmark motor symptom that an estimated 90% of patients with the neurodegenerative disorder…