Margarida Maia, PhD, science writer —

Researchers in the U.S. have created the first brain-like organoids, or mini-brains, that incorporate a working blood-brain barrier (BBB), which is the membrane that regulates which circulating substances can reach the brain and spinal cord, and that typically challenges the entry of medications. Called a BBB assembloid, the human model…

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…

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…

Prilenia Therapeutics plans to submit a marketing authorization application (MAA) by mid-year asking for approval in the European Union of pridopidine, an oral small molecule for treating  Huntington’s disease. The planned filing with the Committee for Medicinal Products for Human Use, part of the European Medicines Agency (EMA),…

Rumi Scientific has joined with X-Chem to advance in early development a suppressor, or inhibitor, of bromodomain-containing protein 9 (BRD9) as a possible Huntington’s disease treatment. “BRD9 inhibition is a novel target for the treatment of HD [Huntington’s disease] identified by our proprietary high…

People carrying fewer Huntington’s disease-causing CAG repeats in the HTT gene — who traditionally are thought to have milder disease — show similar cognitive deficits to those with more CAG repeats, a study found. However, patients with fewer repeats are less likely to show Huntington’s characteristic involuntary muscle contractions,…