New injection for rare protein disease passes first safety check

This early-stage study tested a new drug called ALN-TTRSC04 in 96 healthy people to see if it is safe and how the body processes it. The drug aims to lower a harmful protein that causes transthyretin amyloidosis, a serious disease affecting nerves and the heart. Participants received a single injection, and researchers monitored side effects and drug levels in the blood.