Atalanta Therapeutics has raised $97 million to support planned Phase 1 clinical trials of its experimental RNA interference (RNAi)-based therapies for Huntington’s disease and a genetic form of epilepsy. Each treatment aim to silence the activity of genes that underlie these diseases. Atalanta plans to submit investigational…
Subtle biomarkers of neurodegeneration are evident decades before the onset of overt Huntington’s disease symptoms in people with disease-causing mutations, a study found. Somatic CAG repeat expansions — a phenomenon in which Huntington’s-causing mutations grow, or expand, during a person’s lifetime — were associated with these neurodegenerative biomarkers.
In specific brain cells, the mutation that causes Huntington’s disease grows more and more unstable as years go by, according to a study by U.S. researchers that offers new clues into the generally late onset of the neurodegenerative condition. The findings help to explain why people with Huntington’s don’t…
Abnormalities in a protein called TDP-43, which is known to play a key role in the development of amyotrophic lateral sclerosis (ALS), may also underly Huntington’s disease, a study found, meaning strategies being explored as treatments for ALS might also be useful for treating Huntington’s. “Drugs developed to…
Throughout 2024, the team at Huntington’s Disease News covered the latest developments in research and news related to Huntington’s disease. Here we’ve compiled a list of the top 10 most read stories, each with a brief summary. We look forward to continuing to serve the Huntington’s community throughout…
The U.S. Food and Drug Administration (FDA) has agreed that results from two ongoing Phase 1/2 clinical trials, compared with an external control group of untreated patients, might be sufficient to support an application seeking accelerated approval of AMT-130 for Huntington’s disease, the treatment’s developer said. Key measures…
Beta-blockers, a class of medicines often used to manage heart and blood pressure issues, may slow the onset and progression of Huntington’s disease, according to data from Enroll-HD, the world’s largest observational study in Huntington’s. “Given that there are no disease-modifying agents for HD [Huntington’s disease], the possibility that…
Sarepta Therapeutics has entered a global licensing and collaboration agreement with Arrowhead Pharmaceuticals covering Arrowhead’s therapy candidates, including ARO-HTT, an experimental treatment for Huntington’s disease. Sarepta will obtain exclusive global rights to ARO-HTT and other preclinical programs, as well as clinical and discovery-stage programs for rare genetic…
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…
Ingrezza (valbenazine), an approved treatment for chorea, or uncontrolled movements, related to Huntington’s disease, shows similar effectiveness among patients irrespective of factors like sex, age, or disease severity, according to a new analysis of data from a Phase 3 clinical trial. Moreover, a separate data analysis from…
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