Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Significant news for the Huntington's disease community this week, as the USA's drug regulator, the FDA, has formally approved Austedo, also known as deutetrabenazine, for prescribing in HD. This modified form of tetrabenazine helps control chorea, the jerky movements often found in HD patients, but is taken twice rather than three times a day.
A recent press release from Teva Pharmaceuticals has the HD community excited, claiming "Pridopidine Demonstrates Slowing of Progression of Huntington Disease in PRIDE-HD Study". What's pridopidine, and what can we really say about HD progression in patients treated with it?
A relatively new technology called exome sequencing has identified a few families with novel mutations in their HD genes. These are different than the mutation that causes HD, but allow researchers to better understand the normal role of the HD gene.
A scientific paper declares positive results for a trial of deutetrabenazine in Huntington's disease, but the headlines report the FDA has rejected the drug. Confusing stuff! The reality is positive overall for this new way of treating uncontrollable movements in HD, but patience will be needed to see where it all ends up.
Everyone with Huntington's disease has inherited the same type of mutation from their mother or father, an extra-long repetitive stretch of the sequence C-A-G in their HD gene. But the length of the mutation varies between individuals, and longer repeats are associated with earlier onset of symptoms. A huge new international study reveals that slightly longer-than-normal CAG stretches are much more common than we thought. Surprisingly, this turns out to be good news.
Common depictions of HD emphasizing only its movement symptoms paint an incomplete picture of the real disease. HD causes both motor and non-motor symptoms that, together, affect the entire body. Now, scientists are using a broader lens to explore this full set of HD symptoms and determine how symptoms might be related in the disease.
In early December, Raptor Pharmaceuticals released clinical trial results evaluating a drug called cysteamine in Huntington’s disease. News headlines about this trial are heavy on media spin, and so HDBuzz is here to break down what these new results really mean for the Huntington's community.
The family and friends of individuals with HD often tell doctors that they began to notice changes in behavior long before a diagnosis was made. To better understand these early signs, researchers analyzed a psychological questionnaire filled out yearly for a decade by thousands of HD mutation carriers and their companions. The companions were more likely to perceive worsening symptoms over time.
Today brings news that the first Huntington's Disease patients have been successfully dosed with gene silencing drugs targeting the HD gene. These brave volunteers are the first HD patients to ever be treated with drugs designed to attack HD at its root cause, a treatment approach with huge potential. What about this news has us so excited?