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When young, healthy progenitors of neuron-supporting cells called glia are transplanted into the adult brain, they can grow and replace diseased glial cells, including those carrying a Huntington’s disease-causing mutation, a study in mice reports. “These findings have strong therapeutic implications, as they suggest that in the adult human…

Huntington’s disease could develop at earlier ages in people using calcium channel blockers to control hypertension, a study that looked into genes encoding targets of blood pressure-lowering medications reported. Findings may have important implications for managing high blood pressure in people at risk of Huntington’s, identified before disease…

PTC Therapeutics’ Huntington’s disease treatment candidate PTC518 is well-tolerated and lowers blood levels of the huntingtin protein (HTT) in people with the neurodegenerative disorder. That’s according to interim three-month data from the Phase 2a PIVOT-HD trial (NCT05358717), which found that use of the investigational oral therapy did not…

Scientists have figured out the first molecular events in the formation of toxic huntingtin protein clumps in Huntington’s disease. The findings shed light about why only mutations that result in at least 36 repeats of glutamine (one of protein’s building blocks) in the huntingtin protein cause the disease —…

A computer-based test called SelfCog, which tests various cognitive functions in a standardized manner, yielded promising results in assessing cognitive decline in people with early-stage Huntington’s disease, a study has found. SelfCog demonstrated sensitivity to detect cognitive decline over a one-year follow-up, outperforming traditional cognitive assessments, and showed associations…

Sarah Tabrizi, MD, PhD, a researcher at the University College London (UCL) in the U.K., is the winner of this year’s Lund University Arvid Carlsson Award for her work in understanding Huntington’s disease and developing treatments to slow or stop its progression. The prize is given to researchers who…

The success of CDNF — fully, cerebral dopamine neurotrophic factor — in improving motor coordination in a mouse model of Huntington’s disease supports this protein’s development as a potential therapy for the rare disorder, according to a new study. In the study, CDNF was injected into the brain of…

The oral therapy valbenazine significantly reduces involuntary muscle contractions, or chorea, and patient-reported disease burden in people with Huntington’s disease, according to results from the Phase 3 KINECT-HD clinical trial. Top-line data were announced by Neurocrine Biosciences, the therapy’s manufacturer, in 2021. The full findings have…

Austedo XR, an extended-release oral formulation of Austedo (deutetrabenazine), is now commercially available in the U.S. as a treatment for adults with chorea, or uncontrolled muscle contractions, associated with Huntington’s disease. The new formulation is available in tablet strengths of 6 mg, 12 mg, and 24 mg,…

Note: This story was updated May 16, 2023, to correct the duration of ANX005 infusions. Only the first loading dose of the therapy is given for 21 hours, with subsequent doses having a duration of four to five hours. The experimental therapy ANX005 safely leads to stabilization or improvements in…