Active substance

Results for the substance vutrisiran

1 result

About this active substance

Vutrisiran is a medication used for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR), a rare genetic disease that affects multiple organs and tissues, including the heart, nervous system, and gastrointestinal tract. It is a small interfering RNA (siRNA) that works by reducing the production of transthyretin (TTR), a protein that, in its mutant form, accumulates in the body and causes the symptoms of the disease.

Vutrisiran is administered via subcutaneous injection, usually once every three months, which is a significant advantage for patients compared to other treatments requiring more frequent administration.

Common side effects include injection site reactions, fatigue, headaches, and nausea. In rare cases, severe adverse reactions such as allergic reactions or liver function impairment may occur.

Patients receiving vutrisiran should undergo regular monitoring for liver function and disease progression. The medication represents an innovative therapeutic option for patients with hATTR, helping to reduce symptoms and improve their quality of life.