Progeria is an ultra-rare, fatal, rapid-aging disease. Without lonafarnib treatment, all children with Progeria die of the same heart disease that affects millions of normally aging adults, but at an average age of just 14.5 years. Our recent research endeavors have brought profound scientific progress to Progeria research, including breakthroughs in gene therapy and drug development which could one day translate into major therapies and even the cure.
The Progeria Research Foundation (PRF) was founded in 1999 in response to the complete lack of progress being made to help children with Progeria. Since that time, PRF has been the driving force behind the Progeria gene discovery and the first-ever Progeria drug treatment. PRF has developed programs and services to aid those affected by Progeria and the scientists who conduct Progeria research. Today, PRF is the only non-profit organization solely dedicated to finding treatments and the cure for Progeria. PRF is hailed as a prime example of a successful translational research organization, moving from creation, to gene discovery, to first-ever FDA approval for a Progeria drug, to breakthrough research toward the cure, in just 21 years.
PRF is an official charity of the Boston Marathon!