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…

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…

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…

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 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 Phase 1/2 trial evaluating Wave Life Science’s investigational therapy WVE-003 for Huntington’s disease has started dosing patients, the company has announced. The SELECT-HD trial (NCT05032196), underway in clinical sites in Australia, Germany, Poland, and the U.K., is currently looking to enroll 36 Huntington’s patients, ages…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…