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…
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…
Folia Health is expanding the use of its patient-centered Folia app, designed to collect and manage patient-reported health data, to people with Huntington’s disease (HD). The free app lets patients with certain conditions — now including known or suspected Huntington’s — to record and visualize information about their…
The benefits of Ingrezza (valbenazine) for people with Huntington’s disease — reductions in uncontrolled movements and gains in clinician- and patient-reported health — were seen two weeks after starting treatment and maintained for up to three months, according to new KINECT-HD trial analyses. Ingrezza was cleared last month…
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