Testing a preventive medication for a rare heart and nerve disease

This study is testing a medication called acoramidis to see if it can prevent a serious disease called transthyretin amyloidosis (a genetic condition that damages the heart and nerves) in people who carry the genetic mutation but haven't developed symptoms yet. The medication works by stabilizing a protein in your body that would otherwise misfold and cause damage. Half of participants will receive the medication, and half will receive a placebo (inactive pill), so researchers can see if the medication truly makes a difference.

Currently, there is no proven way to prevent this disease in people who carry the genetic mutation but are still asymptomatic. This trial exists to find out whether starting this medication early—before symptoms appear—can stop or delay the disease from developing entirely.