Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
An enzyme called KMO, which alters the balance of harmful and protective brain chemicals, has been a focus of research into possible Huntington's disease treatments for several years. Now, the first positive results of a KMO inhibiting drug have been published in the scientific journal Cell. HDBuzz investigates...
The mutant huntingtin protein forms clumps, or aggregates, in brain cells. Many scientists believe these clumps contribute to the death of these cells and symptoms in HD. Now scientists have used a beam of neutrons to study the earliest structures formed in these aggregates.
Many new drugs fail because they can’t get into the brain – a major hurdle to developing new Huntington’s disease treatments. Now, researchers have used exosomes – tiny bubbles produced naturally by some cells, to 'float' gene silencing drugs across the blood-brain barrier.
Trials of new treatments for Huntington's disease are happening, but proving whether a drug works requires lots of suitable volunteers. Surprisingly, finding enough volunteers is often difficult. Now a group of HD professionals has shown that education and outreach to patients in the community works well for boosting recruitment.
Weight loss, change in appetite and other metabolic abnormalities are major issues in Huntington's disease. Now, a Swedish team of researchers has used genetically engineered viruses, and some fat mice, to reveal the crucial role of a tiny brain region called the hypothalamus in the metabolic problems of HD.
By popular request, a special feature article on the often confusing topic of 'intermediate alleles' and 'reduced penetrance' – the genetic 'gray area' that frequently comes up in discussions around genetic testing for Huntington's disease.
Dimebon, an experimental drug marketed by Medivation, fails to improve the symptoms of Huntington’s disease patients in the HORIZON trial. This is the end of the road for developing this drug for HD.
NeuroSearch, the developer of experimental Huntington's disease drug Huntexil, has reported on their meeting with the FDA. The FDA requires that another trial be conducted before Huntexil could be approved in the US.
Scientists have successfully used viruses to deliver genes to the brains of Parkinson’s Disease patients. The gene carried by the viruses improved the movement symptoms of patients receiving injections. This proves that gene therapy in the brain can work, providing hope for similar therapies in HD.