Steve Bryson, PhD, science writer —

An investigational oral therapy from PTC Therapeutics called PTC518 safely and effectively reduces levels of the mutant huntingtin protein (mHTT) in Huntington’s disease patients, while being linked to trends of slower disease progression. That’s according to one-year interim data from the ongoing Phase 2a PIVOT-HD clinical trial…

Scientists at the Broad Institute of MIT and Harvard have engineered a harmless adeno-associated virus (AAV) that can efficiently reach the brain, potentially improving the efficacy of brain-targeted gene therapies for neurological conditions such as Huntington’s disease. Current AAVs that deliver gene therapies to cells in the body via…

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.

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…

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…

Neurologists in the U.S. are optimistic about the potential for a new treatment for Huntington’s disease, but emphasize the need for therapies that halt nerve degeneration before symptom onset, according to a report by Spherix Global Insights. Most of the specialist physicians surveyed hoped for a gene therapy…

Forms of mutant huntingtin (mHTT) protein that don’t clump initially drive early disease progression in people with Huntington’s disease, according to a study wherein patient-derived cells were transplanted into newborn mice. These soluble forms of mHTT were found to cause structural changes in the neurons producing them and to…

People with Huntington’s disease were found to have a widespread deficiency of selenium — a trace mineral found naturally in foods such as seafood and Brazil nuts — in their brains, revealing a new potential therapeutic target, according to researchers. Alterations in the levels of other biologically active…

The U.S. Food and Drug Administration (FDA) will review Neurocrine Biosciences’ application of a new oral granule formulation of Ingrezza (valbenazine) for involuntary muscle contractions, or chorea, in adults with Huntington’s disease. The therapy was recently approved in the U.S. for Huntington’s-associated chorea in the form…

Increased levels of the stress response protein MTF1 suppressed the toxic effects of the mutant huntingtin (mHTT) protein — the underlying cause of Huntington’s disease — in cellular and animal models of the neurodegenerative condition, a study showed. Specifically, MTF1 was found to counteract mHTT-associated oxidative stress, a type…