The experimental oral medication branaplam (LMI070) has been granted fast track designation by the U.S. Food and Drug Administration (FDA) as a potential treatment for Huntington’s disease, according to an announcement from the therapy’s developer, Novartis. This designation is designed to speed the development and review of…
News
AMT-130, an investigational gene therapy for the treatment of Huntington’s disease, was overall well-tolerated up to a year after treatment in the first patients to receive the therapy in a clinical trial. The new data, announced by AMT-130’s developer uniQure, cover the first four participants in a Phase…
Treatment with valbenazine significantly lessened chorea — a motor symptom characterized by jerky, unpredictable, and involuntary movements — in people with Huntington’s disease in the Phase 3 clinical trial KINECT-HD, according to an announcement from the therapy’s developer, Neurocrine Biosciences. Neurocrine now is planning to submit an…
The Huntington Study Group is seeking 30 patients to join KINECT-HD2, an open-label study of oral valbenazine capsules for chorea, or jerky involuntary movements, in Huntington’s disease, a press release states. KINECT-HD2 is an extension study of KINECT-HD (NCT04102579), a Phase 3 clinical trial that tested…
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 Needed…
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…
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