Sage Therapeutics is discontinuing the development of dalzanemdor after the investigational therapy failed to improve cognitive abilities relative to a placebo among people with Huntington’s disease enrolled in a Phase 2 clinical trial. Statistically significant gains in cognition with treatment was the main goal of the…
Alnylam Pharmaceuticals has launched a Phase 1 clinical trial to test the safety and pharmacological properties of its treatment candidate ALN-HTT02 in adults with Huntington’s disease, the company announced as part of a financial update. “We [have] made great strides with our pipeline, … initiating a Phase 1…
Higher levels of an enzyme called GST02 in the most affected neurons in Huntington’s disease could underlie early increases in the brain-signaling chemical dopamine that are believed to drive Huntington’s motor symptoms, according to a mouse study. Scientists had previously uncovered that a deficiency in a signaling pathway called…
Diminished production in the brain of a protein known as CHCHD2 may contribute to early neurodevelopmental changes that go on to drive Huntington’s disease, a study reports, with scientists poising the gene responsible for its production as a possible therapeutic target. CHCHD2 is involved in maintaining the workings of…
Clumps of the mutant huntingtin protein, which are the known cause of Huntington’s disease, poke holes in the membrane that surrounds and protects cells’ DNA-filled nucleus, according to preclinical research. These protein aggregates had tiny, protruding fibers that damaged the membrane, which is called the nuclear envelope. Lab-grown cells…
The investigational gene therapy AMT-130 is slowing disease progression for people with early-stage Huntington’s disease, as well as lowering levels of neurofilament light chain (NfL), a marker of nerve cell damage. That’s according to two-year data from two Phase 1/2 clinical trials: one in the U.S. (NCT04120493)…
More than three months of treatment with WVE-003 safely leads to significant reductions in mutant huntingtin — the protein that toxically accumulates in Huntington’s disease — for people with early Huntington’s, while preserving levels of the healthy version of huntingtin and trends of slower brain tissue loss and clinical…
The effects of Ingrezza (valbenazine) on Huntington’s disease-associated chorea grew increasingly stronger over the course of the three-month Phase 3 KINECT-HD clinical trial, according to a post-hoc analysis. A post-hoc analysis is one planned and conducted after the trial has been completed. “This post-hoc analysis of the…
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…
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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