The contents of Ingrezza (valbenazine) can be taken from their capsules and crushed before being sprinkled on soft foods or given via a feeding tube to Huntington’s disease patients who have difficulty swallowing whole capsules, a preclinical study showed. A Neurocrine Biosciences’ oral therapy, Ingrezza…
News
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…
Nearly a year of treatment with Neurocrine Biosciences’ Ingrezza (valbenazine) leads to sustained reductions in uncontrolled movements known as chorea in adults with Huntington’s disease, according to interim data from a Phase 3 trial called KINECT-HD2. “The 50-week data from the ongoing KINECT-HD2 study provide insight on the…
A four-week titration kit for starting on Austedo (deutetrabenazine) — an approved therapy for chorea, or involuntary muscle contractions, due to Huntington’s disease — has so far been well-received by patients in a Phase 4 clinical trial. Titration, in medicine, means starting a drug at a low dose…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to SAGE-718, Sage Therapeutics’ experimental oral therapy for Huntington’s disease. The FDA gives this designation to therapies that have the potential to treat rare diseases, which are defined as conditions that affect fewer than 200,000 people…
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…
Blocking immune system proteins that are part of the body’s complement cascade — which helps the immune system to fight infection — was found to protect nerve cell connections between two brain regions known to be affected in Huntington’s disease in a mouse model of the rare disorder. It…
A lab-made version of a plant enzyme prevented toxic huntingtin protein clumps — that drive Huntington’s disease from forming — in human cell and worm models of the neurodegenerative condition, a study shows. Called stromal processing peptidase (SPP), the enzyme is naturally present in plants’ chloroplasts, where sunlight is…
The recent approval in the U.S. of Ingrezza (valbenazine) for adults with Huntington’s disease-associated chorea means patients now have access to a treatment option that’s likely easier to take, according to the chief medical officer of Neurocrine Biosciences, the therapy’s maker. “Ingrezza is always one capsule,…
Alternative splicing, a molecular process that’s crucial for genes being “read” to produce proteins, is dysregulated in Huntington’s disease from the early stages of neuronal development, a study indicates. Notably, splicing changes were found to be dependent on the length of CAG repeats in the HTT gene — the cause…
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