A Genetic Lottery - a Column by Becky Field

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…

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…

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, “…

The pharmaceutical company Teva is turning to social media to help people with Huntington’s disease (HD) to improve the management of chorea — a hallmark motor symptom that an estimated 90% of patients with the neurodegenerative disorder will develop. The company, fully Teva Pharmaceutical Industries, has established…

ProQR Therapeutics’ experimental therapy QRX-704 significantly reduced levels of the toxic huntingtin (HTT) protein clumps that fuel Huntington’s disease in a mouse model of the disease, a study showed. The therapy, an antisense oligonucleotide (ASO) designed to promote the generation of a shorter, but working version of…

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…

Clumps of protein resulting from HTT mutations, the underlying cause of Huntington’s disease, form distinct structures in different parts of the cell, according to detailed imaging analysis. The findings suggest these clumps form by different mechanisms and may require different therapeutic strategies to block their formation and toxicity that…

The perception of time is impaired in people with symptomatic Huntington’s disease in its early stages — but not in patients without symptoms, a study revealed. These findings suggest that time perception impairment may be used as a clinical tool for assessing those with the neurodegenerative disorder, the scientists…

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…

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…