News

Scientists at the Broad Institute of MIT and Harvard have engineered a harmless adeno-associated virus (AAV) that can efficiently reach the brain, potentially improving the efficacy of brain-targeted gene therapies for neurological conditions such as Huntington’s disease. Current AAVs that deliver gene therapies to cells in the body via…

uniQure expects to begin planned discussions with the U.S. Food and Drug Administration (FDA) in the coming months to outline the future steps for testing its gene therapy candidate AMT-130 for early Huntington’s disease, and seeking regulatory approval of the treatment. In its interactions with the FDA, expected to…

Ayana Bio was awarded $300,000 from the National Institutes of Health (NIH) to sample saffron — the world’s most expensive spice — grown in a lab and identify which of its compounds offer the strongest neuroprotective effects for Huntington’s disease. The Boston-based company will use the grant,…

The U.S. Food and Drug Administration (FDA) has approved Neurocrine Biosciences’ oral granules formulation of Ingrezza (valbenazine) — called Ingrezza Sprinkle — for treating chorea, or involuntary movements, in adults with Huntington’s disease. The new formulation, taken once per day, is designed to provide an easier administration option…

May is Huntington’s Disease Awareness Month, and this year the focus is on the patient experience. Huntington’s disease (HD) is a neurodegenerative disorder thought to affect 4.9 per 100,000 people worldwide, including about 41,000 U.S. residents, and can impact many aspects of everyday life. While each patient’s journey…

Daily treatment with Ingrezza (valbenazine) led to sustained reductions in chorea — characterized by involuntary, unpredictable body movements — for nine months among the small group of Huntington’s disease patients simultaneously using antipsychotic medications in the Phase 3 KINECT-HD2 trial. In addition, improvements in chorea were also…

A type of brain nerve cells called layer 5a pyramidal neurons — especially those projecting into the striatum, a brain region greatly affected in Huntington’s disease — are lost early in the course of Huntington’s, a study found. Excessive CAG repeats in the HTT gene, the cause of…

Scientists at the University of California, Irvine (UCI) have mapped onto a genetic database nearly a million tandem repeat (TR) expansions — head-to-tail copies of short DNA sequences — to better understand how they’re linked to several human diseases, including Huntington’s. This work, the team says, may provide “critical…

Cariprazine, an oral medication approved for schizophrenia, may ease some motor symptoms people with Huntington’s disease experience across different stages of the condition, a small study suggests. How cariprazine works in Huntington’s still isn’t clear, but researchers think it may balance out dopamine levels when they’re too high, as…

Small molecules developed by Design Therapeutics were able to reduce the activity of the disease-causing mutant gene in preclinical models of Huntington’s disease, according to an announcement from the company. “We are highlighting an exciting new program for Huntington’s Disease, an indication of tremendous unmet medical need,”…