Tag: Sirna

ONPATTRO (patisiran) was the first-ever small interfering RNA (siRNA) therapeutic approved by the U.S. Food and Drug Administration (FDA) in 2018 for treating a genetic disease. This groundbreaking treatment, which works by "silencing" a gene, has raised a crucial question in the field of genetic medicine: is ONPATTRO a gene therapy?

In 2018, ONPATTRO became the first FDA-approved medication using RNA interference technology, ushering in a new era of gene-based medicines. The groundbreaking therapy acts as a gene silencer, specifically targeting and reducing the production of a protein responsible for a rare, debilitating genetic disease known as hATTR amyloidosis.

In clinical studies, some patients experienced a drop in LDL cholesterol levels within just two weeks of their first Leqvio dose. Understanding how long does it take Leqvio to work is key for managing expectations and adhering to the unique treatment schedule that offers a long-lasting effect.