Tag: Hattr pn

According to clinical trial data, AMVUTTRA begins decreasing transthyretin (TTR) protein levels just weeks after the first dose. However, it may take several months to see clinical improvements in symptoms, as the treatment gradually works to slow disease progression and can even reverse some symptoms over the long term.

According to the results of the Phase 3 HELIOS-A clinical trial, 48% of patients treated with AMVUTTRA experienced a reversal of neuropathy impairment from their baseline after 18 months, a significant improvement compared to the placebo group. For many suffering from this progressive and debilitating condition, the key question remains: Does AMVUTTRA really work?

Hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) is a rare and progressive genetic disorder, estimated to affect between 10,000 and 40,000 people globally. This condition is caused by a gene mutation that results in abnormal protein deposits that damage the nervous system and other vital organs.