Patient Community Stories

When Sophia first learned she had tested gene positive for Huntington’s disease, she was devasted. Sophia ultimately decided that she would not let HD define her; she chose to fight back by making positive changes like pursuing therapy and deeply engaging with the HD community. She finds strength and a sense of purpose in being an advocate, refusing to feel helpless, and having a positive effect by supporting research, keeping informed, and fostering connection within the community. ​“I was scared of Huntington’s disease my whole life. And now I’m full of hope, and positive, because I know that science is developing fast.”

Learn more about Sophia’s HD journey below. 

Ashley’s father was diagnosed with Huntington’s disease when she was 15 years old; she and her brother became their dad’s primary caregivers as his symptoms progressed. She remembers finding very little information online at the time about HD and support for caregivers. She shares her story as an HD advocate to help others struggling with the challenges facing HD families. ​“I personally can’t find a treatment and I can’t find a cure – but I have a voice, and I realized that I have the ability to help people in another way.”

Learn more about Ashley’s experience as a caregiver below.
 

Ron was one of the first participants in our EtranaDez clinical trial in hemophilia B—and one of the early patients to be dosed with any gene therapy for this disease. He hits the gym often, lifting weights and doing other strength-training exercises.