May is Huntington’s Disease Awareness Month, and this year the focus is on the patient experience. Huntington’s disease (HD) is a neurodegenerative disorder thought to affect 4.9 per 100,000 people worldwide, including about 41,000 U.S. residents, and can impact many aspects of everyday life. While each patient’s journey…
Daily treatment with Ingrezza (valbenazine) led to sustained reductions in chorea — characterized by involuntary, unpredictable body movements — for nine months among the small group of Huntington’s disease patients simultaneously using antipsychotic medications in the Phase 3 KINECT-HD2 trial. In addition, improvements in chorea were also…
A type of brain nerve cells called layer 5a pyramidal neurons — especially those projecting into the striatum, a brain region greatly affected in Huntington’s disease — are lost early in the course of Huntington’s, a study found. Excessive CAG repeats in the HTT gene, the cause of…
Scientists at the University of California, Irvine (UCI) have mapped onto a genetic database nearly a million tandem repeat (TR) expansions — head-to-tail copies of short DNA sequences — to better understand how they’re linked to several human diseases, including Huntington’s. This work, the team says, may provide “critical…
Cariprazine, an oral medication approved for schizophrenia, may ease some motor symptoms people with Huntington’s disease experience across different stages of the condition, a small study suggests. How cariprazine works in Huntington’s still isn’t clear, but researchers think it may balance out dopamine levels when they’re too high, as…
Small molecules developed by Design Therapeutics were able to reduce the activity of the disease-causing mutant gene in preclinical models of Huntington’s disease, according to an announcement from the company. “We are highlighting an exciting new program for Huntington’s Disease, an indication of tremendous unmet medical need,”…
Prilenia Therapeutics plans to submit a marketing authorization application (MAA) by mid-year asking for approval in the European Union of pridopidine, an oral small molecule for treating Huntington’s disease. The planned filing with the Committee for Medicinal Products for Human Use, part of the European Medicines Agency (EMA),…
Two types of orally available small molecules ameliorated symptoms and slowed progression of Huntington’s disease in a mouse model, a study reported. The treatments effectively reduced the levels of the mutant huntingtin protein in the brain without affecting normal huntingtin. The mutant version causes Huntington’s, a neurodegenerative condition.
A first clinical trial of SKY-0515, an experimental treatment for Huntington’s disease, is starting to give multiple ascending doses to healthy adults randomized to the therapy rather than a placebo, Skyhawk Therapeutics, its developer, announced. Initiation of this part of the Phase 1 trial (ACTRN12623001161617), taking place in…
The brain of people with pediatric-onset Huntington’s disease show impaired transport of blood sugar, or glucose, and low levels of a cellular machinery involved in glucose metabolism, a small study showed. Many of these abnormalities were observed in comparisons not only with adults without Huntington’s, but also with people with…
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