Antisense Therapy To Treat Huntington’s Disease and Spinocerebellar Ataxia
Scott Schobel, MD, chief medical officer at Vico Therapeutics discussed the latest data testing an antisense therapy to treat Huntington’s disease and spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3).
Huntington's disease is a rare neurodegenerative disorder characterized by chorea movements, behavioral and psychiatric disturbances, and dementia. The gene involved is the huntingtin gene in which a CAG expansion occurs that results in neuronal damage, especially in the basal ganglia. SCA1 and SCA3 are rare neurological disorders, mostly involving the cerebellum, and the underlying genetic abnormalities also include CAG expansions.
VO659 is an antisense oligonucleotide investigational therapy designed to target the CAG repeat expansion. It is being developed to treat Huntington’s disease as well as SCA1 and SCA3. Recently, the company announced they have started enrolling patients for a phase 1/2 clinical trial. It is a multi-center, open-label basket study designed to assess the safety and tolerability of multiple ascending doses of VO659 administered intrathecally in participants with early manifest HD or mild to moderate SCA1 or SCA3. The study is expected to enroll approximately 71 participants.
Смотрите видео Antisense Therapy To Treat Huntington’s Disease and Spinocerebellar Ataxia онлайн без регистрации, длительностью часов минут секунд в хорошем качестве. Это видео добавил пользователь CheckRare 23 Июнь 2023, не забудьте поделиться им ссылкой с друзьями и знакомыми, на нашем сайте его посмотрели 39 раз и оно понравилось людям.