News

A Phase 1/2 clinical trial testing uniQure’s potential gene therapy AMT-130 in people with Huntington’s disease can continue enrollment, according to a positive review by the study’s independent data safety monitoring board (DSMB). The DSMB reviewed safety data from four patients given the higher dose of the therapy, which…

The U.S. Food and Drug Administration (FDA) has given fast track designation to Prilenia Therapeutics’ pridopidine as a potential oral treatment for Huntington’s disease. This designation is given to therapies that show considerable potential in addressing serious conditions for which available treatments fall short. It is meant to…

Celebrities turned out in force — albeit virtually — to support the 7th Annual Freeze HD event, hosted by the Huntington’s Disease Society of America (HDSA), which raised more than $211,000 to help improve the lives of families affected by Huntington’s disease (HD). The 2.5-hour show, held online for…

Two scientists investigating Huntington’s disease have been awarded prizes by the Hereditary Disease Foundation, recognizing their leadership in disease research. The Leslie Gehry Brenner Prize for Innovation in Science was awarded to Elena Cattaneo, PhD, director of the Laboratory of Stem Cell Biology and Pharmacology of Neurodegenerative Diseases…

A Phase 3 clinical trial investigating the efficacy and safety of oral pridopidine for those with early stage Huntington’s disease is now underway at more than 50 clinical sites in the U.S., the U.K., Canada, and Europe, according to Prilenia Therapeutics, which is developing the treatment candidate. Prilenia…

Frequent bladder, bowel, and sexual problems were reported by people with Huntington’s disease, a study reveals. Recommended Reading October 13, 2021 Columns by B.J. Viau These 5 Changes Are…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

The U.S. Food and Drug Administration (FDA) has granted an orphan drug designation to SOM3355, an investigational treatment for chorea, or involuntary jerky movements, associated with Huntington’s disease, its developer SOM Biotech has announced. Orphan drug status helps to encourage the development of therapies for rare…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

NeuExcell Therapeutics has entered into a research deal with Spark Therapeutics to advance the development of a safe and effective neuroregenerative gene therapy for Huntington’s disease. Under the terms of the agreement, Spark, a member of the Roche Group, will have access to NeuExcell’s gene therapy platform…