News

Wave Life Sciences is making plans to launch a potentially pivotal Phase 2/3 clinical trial in the U.S. to test its therapy candidate WVE-003 in people with Huntington’s disease. Pivotal trials are those whose data, if positive, are meant to be used to support applications seeking a treatment’s…

One-year results from the Phase 2 PIVOT-HD clinical trial testing PTC Therapeutics’ Huntington’s disease treatment PTC518 are expected in the second quarter. That’s according to an update from PTC, which also noted that in a December meeting with the U.S. Food and Drug Administration (FDA), the agency…

LETI-101, an experimental gene-editing therapy for Huntington’s disease, showed promising proof-of-concept effects in mouse and cell models by reducing levels of the disease-causing mutant huntingtin (HTT) protein, while preserving HTT’s healthy version. In nonhuman primates, the therapy showed good safety and distribution across brain regions profoundly impacted by Huntington’s. The…

Specific genes involved in repairing mismatched DNA contribute to the development of Huntington’s disease by driving fast somatic CAG repeat expansion — a phenomenon in which Huntington’s-causing mutations grow, or expand, during a person’s lifetime, scientists report. Their study, done in a mouse model of Huntington’s, showed that targeting…

While the vast majority of Huntington’s disease patients in North America have the uncontrolled and jerky movements known as chorea at clinically relevant levels, about a third of these people are getting medications specifically for this motor symptom. Among treated patients, about half are prescribed VMAT2 inhibitors, the…

Pediatric-onset Huntington’s disease is more severe than its adult-onset form, with faster and more extensive shrinkage of brain regions involved in movement, an imaging study has found. Atrophy, or a loss in brain volume, “was remarkable and more severe” in patients with pediatric-onset disease compared with adult-onset Huntington’s, the…

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…