EHDN just held its plenary meeting in Vienna; over 1,000 delegates attended. The event confirmed the pace of HD research is accelerating and that was underscored by pharmaceutical company Roche announcing their pivotal ‘huntingtin lowering therapy’ trial of RG6042 (formerly IONIS Httrx). They currently anticipate the trial will recruit up
Press Releases
Potential Huntington’s treatment gets priority designation
The European Medicines Agency (EMA) granted PRIME designation for the Huntingtin-lowering (sometimes called gene silencing) antisense drug, known as HTTRx or RG6042, for the treatment of people with Huntington’s disease. This means that the EMA supports the development of the drug to target an unmet need, speeding up evaluation so
New animal models of HD are leading to a deeper understanding
We are still relatively in the dark about why HD affects certain parts of the brain more than others and scientists have been working on a new animal model which they hope will help deepen our understanding of the reasons. Thanks to HD Buzz for this very readable article. https://en.hdbuzz.net/260
Scottish Medicines Consortium Announcement on Spinraza
The Scottish Medicines Consortium are the body who authorise the use of new drugs in NHS Scotland. They have been evaluating a new drug used to treat Spinal Muscular Atrophy, an inherited condition which attacks the motor neurones in the spinal cord, causing muscle wasting. The new drug, called Spinraza,
How close are we to a cure for Huntington’s Disease?
Cracking article exploring the journey towards a treatment for Huntington’s disease. Treatment is always the word we prefer; it’s much more realistic than cure. A cure is likely to depend on technologies that are much further away than the current gene silencing drugs in development. These drugs, if successful, will
Managing Expectations About HD Breakthrough
The historic news of a breakthrough in the treatment of Huntington’s disease (HD) has caused excitement worldwide, and for good reason. It is the first time any drug has lowered the levels of the faulty protein known to cause the disease and consequently, it is hoped, will improve the devastating