Managed Healthcare Executive November 9, 2021
The Pfizer and Moderna COVID-19 vaccines introduced much of the world to ribonucleic acid (RNA), which has long played second fiddle to DNA. But in addition to the messenger RNA vaccines, treatments targeting and using RNA in a variety of ways are filling the pipeline and exciting researchers and drug developers.
When her toddler was diagnosed with progeria in 1998, Leslie Gordon, M.D., Ph.D., faced three distinct challenges: Progeria was extremely rare. It was incurable. And most devastatingly, it was fatal.
Gordon’s son, Sam Berns, had appeared healthy at birth, but by the time he turned 2, it was clear that something was wrong. Caused by a mutation in the LMNA gene, progeria leads to rapid aging and physical changes,...