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…
The Huntington’s Disease Society of America (HDSA) has awarded grants totaling more than $2.1 million to support its Centers of Excellence, which include multidisciplinary care teams with a track record of providing exemplary care for people with Huntington’s disease and their families. A total of 57 institutions…
Excessive CAG repeats, the genetic defect that causes Huntington’s disease, lead to processing errors and the production of unexpected proteins that form clumps and promote nerve cell death, a new study found. This discovery, according to the researchers, sheds new light on the abnormal toxic protein production seen in…
Eating a high-fiber diet led to improvements in memory and reductions in depression-like behavior in a mouse model of Huntington’s disease, a new study reports. “What we found is very exciting. For the first time we’ve shown that high-fiber intake not only enhanced gastrointestinal function, it also improved cognition…
A newly launched company known as HD Immune, based in Vienna, is working to develop an antibody treatment for Huntington’s disease. The potential therapy showed promise in a mouse model, according to the study, “Reducing huntingtin by immunotherapy delays disease progression in a mouse…
In a biomarker study, scientists in South Korea found that levels of two proteins — neurofilament light chain (NfL) and phosphorylated tau (p-Tau) — serve as markers to indicate disease progression in Huntington’s. Their findings on p-Tau are novel, and show that levels of this protein — known to…
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