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Discover all available medicines and treatments
Brand: Amvuttra
Vutrisiran is a double-stranded small interfering ribonucleic acid (siRNA) that targets and silences the transthyretin (TTR) gene, thereby reducing the production of abnormal TTR protein. It is used to treat the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. This is a rare, progressive, and life-threatening disease caused by the buildup of misfolded TTR protein in various tissues and organs, including the nerves and heart.
Vutrisiran is used to treat the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Outcome:
Increased risk of liver damage
Mechanism:
Additive hepatotoxicity
Outcome:
Increased risk of gastrointestinal bleeding
Mechanism:
Additive effects on platelet function
Outcome:
Reduced absorption of vutrisiran
Mechanism:
Changes in stomach pH
Most likely new formulation: Subcutaneous self-administration version (Year: 2025, 70% confidence)
Based on clinical trial data and current usage trends, there is a high likelihood (85%) that vutrisiran will remain a key treatment option for hATTR amyloidosis.
RNA Interference Therapeutic
siRNA