I’m kicking off a series on HOPE with a positive video from Post Card from Palm Springs, “Highlights of the 2019 CHDI Therapeutics Conference” with Charles Sabine, Huntington’s Disease Advocate. Watch the video here:
At this time, the Huntington’s disease community is filled with HOPE because there are many promising clinical trials being conducted around the world. i.e. Roche RG6042 https://clinicaltrials.gov/ct2/show/NCT03842969, Uniqure AMT-130 https://en.hdbuzz.net/274, Vaccinex vaccinex-signal-trial/, Wave Life Science wave-life-sciences-provides-timing-update-precision-hd-clinical.
Huntington’s disease (HD) is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It deteriorates a person’s physical and mental abilities during their prime working years and has no cure. HD is known as the quintessential family disease because every child of a parent with HD has a 50/50 chance of inheriting the faulty gene. Today, there are approximately 30,000 symptomatic Americans and more than 200,000 at-risk of inheriting the disease. http://www.hdsa.org
Mai-Lise Nguyen, Roche Patient Partnership Director says the trial will answer these question, “Does the lowering of mutant huntingtin translate into actual clinical benefit? Is it a meaningful benefit for patients and their family?”
Approximately 660 people are enrolled in the clinical trial in 15 countries around the world and we all await the results from the trial.