A Genetic Lottery
— Rebecca Field

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…

Nonprofits around the world are hoping to “turn up the global volume” for Huntington’s Disease Awareness Month, an annual event in May to call attention to the disease, estimated to affect up to 7 per 100,000 people globally. In the U.S., about 41,000 Americans are affected by the disease,…

The Phase 3 PROOF-HD trial failed to meet its main goal of showing that pridopidine, Prilenia Therapeutics’ experimental therapy, could slow functional decline compared with a placebo for adults with Huntington’s disease. However, preliminary top-line data indicated a potential slowing of disease progression among patients who were…

Asklepios Biopharmaceutical (AskBio) has launched a first-in-human clinical trial testing AB-1001 — an experimental gene therapy designed to modulate cholesterol metabolism in the brain — in people with Huntington’s disease. The trial is currently recruiting participants at a site in Paris. It is open to adults, ages 18…

Subtle changes in speech are present before obvious symptoms of Huntington’s disease appear, and could potentially be a quantitative biomarker for the neurodegenerative disorder, a small study found. The changes in speaking identified among Huntington’s patients were linked to age and an individual’s number of disease-causing CAG repeats —…

A Phase 1/2a clinical trial testing VO659, Vico Therapeutics’ experimental therapy, in people with Huntington’s disease and other hereditary neurological disorders caused by a similar type of mutation has dosed the first patient. “We are encouraged by the continued progress of our development program and very pleased to…

The last patient has completed the final visit in the Phase 3 PROOF-HD trial testing oral pridopidine for Huntington’s disease. That means that the study’s main placebo-controlled part is done, according to Prilenia Therapeutics, pridopidine’s developer, which sponsored the nearly 18-month-long trial. Now, the first data are…

The biopharmaceutical UCB and Aitia have joined forces to accelerate the development of a novel Huntington’s disease treatment. The collaboration will harness Aitia’s artificial intelligence (AI) technology to identify new targets and therapies for Huntington’s that will be further validated using UCB’s expertise in treatment research and…