Thank You Thursday, HD-ISS, and Gene Therapy 101

Happy Thank You Thursday from HDSA  

Support from HD families like you is what allows HDSA to fight for a world free of HD. This year, HDSA was overwhelmed by generosity that poured in on #GivingTuesday, with gifts surpassing $100,000. These dollars support community services and education, advocacy and outreach, researchers searching for treatments and cures for HD, and more. Thank you to all who donated, and to all those who support HD research by staying up to date with this blog, participating in clinical trials and online surveys, and more! The more awareness within our community, the greater the research participation, and the faster the field can move towards effective treatments for HD.    

Dr. Sarah Tabrizi discusses HD-ISS on Help 4 HD podcast  

Last week, esteemed HD clinician and researcher, Dr. Sarah Tabrizi, joined Help 4 HD podcast host Lauren Holder for a discussion on her entry into HD research and the HD Integrated Staging System (HD-ISS). This novel, evidence-based classification system for stages of HD encompasses the full course of HD from birth to late stages of the disease. Designed for use in clinical research and trials, this new staging scale for HD could help standardize clinical research and enable study of earlier interventions for HD. To listen to the full episode, click here.  

Gene Therapy 101: New Resource from ASGCT 

Gene therapies are constantly making science headlines. Viral gene therapies, CRISPR, and other research hot topics stir a buzz any time they hit our news feeds. While an exciting evolution for how scientists and medical professionals may be able to treat diseases, gene therapies are tricky and come with nuanced vocabulary that can be difficult to understand. The American Society of Gene and Cell Therapy (ASGCT) recently released a series of educational materials for understanding gene therapies—what are they, how do they work, how are they implemented in clinical trials, and more. Click here to read more.