The August 9th press release from Voyager Therapeutics left many of us scratching our heads. Luckily, HDBuzz swooped in with their latest article that deciphers Voyager’s news and sheds light on the numerous therapies in the HD research pipeline and their unique approaches to treating HD. In short, Voyager is no longer pursuing a gene therapy that was in the works for HD, VY-HTT01, which was scheduled to begin a clinical safety trial for later this year, although no participants had yet been recruited. While this news poses a set-back for the short term, Voyager is refocusing their efforts to develop a new technology that would improve the way gene therapies can be administered for rare diseases like HD. Instead of injection into deep brain tissues, this new delivery method would allow therapies to be delivered by a less-invasive injection into the blood, which could present long-term benefits to the HD community. For the full breakdown on Voyager’s news and other promising therapies in the HD research pipeline, read the full article on HD Buzz’s website here.
Louise Vetter and Lauren Holder Discuss New Online Community for HD Families
In collaboration with PatientsLikeMe (PLM), HDSA recently launched a tailored community for HD families. On August 12, HDSA President & CEO, Louise Vetter, joined host Lauren Holder on an episode of Help 4 HD that walked through all that the platform provides and the blind spot it fills in drug development. PLM is not only a place for families to anonymously come together to find support and share experiences, it’s a place to track day-to-day symptoms, get answers to questions, and find reputable information free from marketing schemes and misinformation. Additionally, PLM is a platform to curate the patient perspective for improved diagnosis, care, and treatment of HD. Click here to join the tailored community today.
ChANGE HD: Research to Improve Our Understanding of the HD Brain
Young people with a parent or grandparent who has tested positive for or been diagnosed with Huntington’s disease are invited to participate in ChANGE HD. This brain imaging study is led by the University of Iowa Hospitals and Clinics and has study sites in Iowa City, Houston, New York, Philadelphia and Sacramento. It focuses on young people ages 6-30 who have a family history of HD. HD is considered an adult-onset disease. However, research has shown that brain changes are evident before motor symptoms begin. In fact, the gene responsible for HD plays a role in brain development and is present throughout the lifespan. With gene knockdown therapy showing promise for treatment of HD, it is essential to understand how removing or disabling the gene could impact brain development. For more information on this study, view the research flyer here, review FAQ’s here, or visit the study website at www.changehd.org.