PTC Therapeutics provides enrollment update 

PTC Therapeutics shared a press release this week to confirm that enrollment is active and ongoing for its Phase 2 PIVOT-HD trial of PTC518, an oral huntingtin-lowering drug. PTC had begun the study, but wanted to make some changes including extending the length of it, and they got approval to do so in several European countries as well as Australia. However, enrollment has been paused in the U.S. right now, while PTC discusses the planned changes further with the FDA and provides additional data. To be clear, this is not because of any known issue with the drug itself – no issues have arisen so far. It is simply an ongoing conversation with regulators to ensure that trial plans proceed as safely as possible. To read the full press release, click here.  

Teva shares good news for long term use of Austedo  

Earlier this week, Teva Pharmaceuticals announced results from the ARC-HD (Alternatives for Reducing Chorea in Huntington’s Disease) trial, an approximately 3-year study testing the safety and tolerability of long-term treatment with AUSTEDO® (deutetrabenazine) tablets for chorea in people with HD. Austedo was approved for HD in 2017, so we already knew it was safe and effective in the short term. In ARC-HD, known as a “long term extension,” every participant took the drug for up to 3 years. It showed that the drug continues to be safe, tolerable, and effectively control chorea. Additionally, on average, when people with HD switched from tetrabenazine overnight, Austedo worked a little better. For the full press release, click here.  

Survey: Family Caregiving in HD Families 

A genetic counseling student at the University of California, San Francisco is working with a group of researchers to explore the experiences of family members who take care of people with Huntington’s Disease. They are interested in understanding how family members who take care of people with Huntington’s Disease (family caregivers) understand and feel about palliative care and genetic counseling. If you are a family caregiver or HD family member, you may be eligible to participate in this 15-25-minute-long survey. Volunteers will be entered into a raffle or a chance to win a $50 Amazon gift card. For more information, please contact Justin Peng (Justin.Peng@ucsf.edu), visit the survey link, or check out HDSA’s page of vetted online surveys.