Roche has discontinued dosing in the Phase 3 GENERATION HD1 trial of its investigational therapy tominersen in adults with Huntington’s disease. The decision was made after a pre-planned review of trial data by an independent data monitoring committee, which issued a “no go” recommendation based on tominersen’s potential benefit/risk…
News
Stalled protein-building may underlie Huntington’s disease, according to data from a recent study. The findings showed that the mutated huntingtin protein, which is at the heart of the disorder, slows down ribosomes, the small cell machinery structures responsible for building new proteins. According to investigators, knowing this and understanding…
Women with Huntington’s disease have worse motor, cognitive, and depressive symptoms than men with the condition, according to data from the global Enroll-HD observational study. Notably, no sex-specific differences were found for age at diagnosis and symptom progression over the four years of follow-up. These findings help to increase…
An alliance of Huntington’s disease (HD) organizations in the U.K. is calling for patient contributions to a project that intends to show the world what it’s like to live with the neurodegenerative disorder. Through the end of May, the “living history” project is collecting photos, poems, or other works…
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…
Wearable devices show considerable promise in helping diagnose people with Huntington’s disease, but the studies evaluating them need to be standardized to maximize their potential benefit to patients, a review found. That review determined that these devices can distinguish people with and without Huntington’s, and between symptomatic and pre-symptomatic…
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…
With some recent additions, the Huntington’s Disease Society of America (HDSA) has expanded its Centers of Excellence program — ensuring comprehensive care provided by multidisciplinary care teams — to 54 sites that have demonstrated expertise and commitment in Huntington’s (HD) care. The program grew from 20 U.S. centers…
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
Recent Posts
- What the holidays mean when living with Huntington’s disease
- $12M grant will back first trial of stem cell therapy for Huntington’s
- A tribute to the kindness of strangers in life with Huntington’s
- How to support someone who is planning for future care
- Antipsychotics improve functional capacity, independence in HD
- Despite dwindling resources, hope endures among researchers
- The ‘invisible’ symptoms of Huntington’s that people don’t see
- Genetic variant delays Huntington’s onset by up to 23 years: Study
- Watching Huntington’s switch roles in our marriage was jarring
- The gift of community is what I’m most thankful for this year