News

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…

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

A few years ago, Shelby Lentz wrote a song called “Champions,” originally inspired by the “Rocky” movies. The plucky country anthem has since taken on a deeper, more personal meaning as she and members of her family battle Huntington’s disease. “We wrote this entire song, and I recorded…

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…