Levels of glial fibrillary acidic protein (GFAP), a marker of damage to specific neuron-supporting cells, in the fluid surrounding the brain and spinal cord increase with the progression of Huntington’s disease, a study showed. Higher GFAP levels in the cerebrospinal fluid (CSF) were also significantly associated with more severe…
Ingrezza Sprinkle, a new formulation of Ingrezza (valbenazine) that’s designed to be easier to swallow, is now available in the U.S, Neurocrine Biosciences, which markets the therapy, announced. Ingrezza Sprinkle comes in capsules that are meant to be opened and the contents sprinkled over soft foods like yogurt,…
The investigational gene therapy AMT-130 is slowing disease progression for people with early-stage Huntington’s disease, as well as lowering levels of neurofilament light chain (NfL), a marker of nerve cell damage. That’s according to two-year data from two Phase 1/2 clinical trials: one in the U.S. (NCT04120493)…
More than three months of treatment with WVE-003 safely leads to significant reductions in mutant huntingtin — the protein that toxically accumulates in Huntington’s disease — for people with early Huntington’s, while preserving levels of the healthy version of huntingtin and trends of slower brain tissue loss and clinical…
Neurocrine Biosciences is offering a new online educational resource called More Than HD that seeks to inspire and give hope to Huntington’s disease patients and their caregivers by helping them prepare for their journey with the disease. “It was important for us at Neurocrine to create More Than…
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…
Significant impairments in cognitive function are evident in people with Huntington’s disease relative to healthy adults, as assessed with a disease-specific battery of cognitive tests, top-line data from a Phase 2 clinical trial show, meeting its main goal. The two-part trial, called SURVEYOR (NCT05358821), also evaluated the safety…
The effects of Ingrezza (valbenazine) on Huntington’s disease-associated chorea grew increasingly stronger over the course of the three-month Phase 3 KINECT-HD clinical trial, according to a post-hoc analysis. A post-hoc analysis is one planned and conducted after the trial has been completed. “This post-hoc analysis of the…
Clinical trials testing either tominersen, an experimental therapy designed to target the underlying cause of Huntington’s disease, or dalzanemdor, an oral candidate that aims to improve cognitive abilities, are currently enrolling Huntington’s patients in the U.S. Updates on the therapies’ clinical programs were provided by representatives of their…
The Huntington’s disease community was encouraged to step up and get involved in observational studies to help scientists better understand the neurodegenerative disorder and accelerate the development of potential treatments. Participating in research “is the greatest thing that we can do to help expedite treatments for HD [Huntington’s disease],”…
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