PROOF-HD, Rare Patient Voice, This Week in HD History

Last Participant Completes the PROOF-HD Study  

This week, Prilenia Therapeutics shared a press release announcing the completion of the last patient visit in the PROOF-HD study, a large trial that is testing a drug called pridopidine in participants with HD. The goal of the study is to understand whether pridopidine can help to preserve people’s day-to-day abilities over the course of about 18 months. Results from the study are expected to be announced in Q2 of 2023. To read more, click here.  

Compensated Research Survey Opportunities Through Rare Patient Voice  

Rare Patient Voice connects patients and caregivers with the opportunity to voice their opinions through paid surveys and interviews to improve medical products and services. There are two current research opportunities for Huntington’s Disease patients and caregivers living within the United States. One is a 25-minute online survey, with $50 compensation; the other is a 5-minute online survey with $200 gift card compensation.  

To participate, you will need to follow this link to sign up with Rare Patient Voice: https://rarepatientvoice.com/hdsa/. Then you will receive an email invite to a screening survey to see if you qualify. You may also receive emails in the future about other opportunities to participate. 

This Week in HD History  

HD is caused by a mutation in the huntingtin gene that leads to the production of mutant huntingtin protein. Mutant huntingtin builds up in the brain and evidence suggests that this contributes to the onset of HD symptoms.  

In 2000, scientists from Columbia University published a mouse model of HD in which expression of mutant huntingtin could be switched on and off. When mutant huntingtin protein was produced, the mice experienced symptoms of HD, but when production of mutant huntingtin was blocked in those same mice, their symptoms of HD subsided. This research was some of the first to support the hypothesis that lowering of mutant huntingtin protein could stop or slow the symptoms of HD. This idea has informed many experimental treatment approaches being pursued by pharma companies today.