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Potential Chorea Treatment Valbenazine Under Review in US

The U.S. Food and Drug Administration (FDA) has agreed to review Neurocrine Biosciences’ supplemental new drug application (sNDA) for valbenazine to treat chorea caused by Huntington’s disease. “This sNDA filing advances our effort to bring a potential new treatment option to the many thousands of people experiencing chorea associated with…

New RNA-targeting Therapy Reduces Toxic HTT Protein Buildup

Researchers have developed a new therapy that reduced the toxic buildup of the mutant huntingtin (HTT) protein — the hallmark of Huntington’s disease — in lab-grown neurons from Huntington’s patients and in a mouse model of the disease. The CRISPR-based therapy was designed to specifically target the HTT gene’s messenger…

Study Finds How Branaplam May Work to Help Treat Huntington’s

Branaplam (LMI070), an experimental oral therapy for Huntington’s disease that’s currently in early clinical trials, reduces levels of the mutant huntingtin protein by interfering with a process called mRNA splicing, a new study reveals. “Here, we delineated the mechanism of action of Branaplam,” researchers wrote. The study, “…