Inotersen, a new drug developed by Ionis Pharmaceuticals, offers hope for patients with hereditary transthyretin amyloidosis (hATTR). hATTR is a rare, progressive, and fatal genetic disorder caused by mutations in the transthyretin (TTR) gene. The disease is characterized by the buildup of abnormal proteins in the body, which can lead to organ damage and death.
Inotersen is an antisense oligonucleotide (ASO) therapy that works by targeting the mutated TTR gene and reducing the production of the abnormal proteins. In clinical trials, Inotersen was found to be effective in reducing the levels of the abnormal proteins in the body, leading to improved symptoms and quality of life for patients.
Inotersen is the first ASO therapy to be approved by the U.S. Food and Drug Administration (FDA) for the treatment of hATTR. It is administered as a once-monthly subcutaneous injection and is available in both a prefilled syringe and a single-use autoinjector.
Inotersen is a welcome addition to the treatment options available for hATTR patients. It offers a new hope for those living with this rare and debilitating disease. It is also an important step forward in the development of treatments for other rare diseases caused by genetic mutations.
The availability of Inotersen is a major breakthrough for hATTR patients and their families. It offers a new hope for those living with this rare and debilitating disease. With continued research and development, it is possible that more treatments will become available in the future, offering even more hope for those affected by hATTR.