Progeria Research Foundation

The Progeria Research Foundation (PRF) was established in 1999 to discover treatments and the cure for progeria and its aging-related conditions, such as heart disease. The foundation has played a critical role in taking children with progeria out of the background, where they had been for over 100 years, and putting them at the forefront of scientific efforts. In just 20 years, the organization has achieved extraordinary progress towards finding a cure for progeria: identifying the progeria gene in 2003, initiating the first-ever clinical drug trials for progeria in 2007, discovering the first treatment for progeria in 2012 as a result of the trial, and achieving FDA approval for the drug (Zokinvy) in 2020. Most recently, several PRF-funded research initiatives in gene editing have shown breakthrough progress in preclinical studies, which we hope will one day lead to clinical trials.

PRF has achieved extensive global awareness of the disease and confirmation of critical biological links between progeria, heart disease and the aging that affects all of us. Through the creation of research-related programs and services, PRF has taken an obscure, ignored disease to global recognition and treatments in a relatively short period of time. PRF has set an example of how a patient organization can successfully drive research and therapy development for a neglected disease, regardless of how rare the disease is. More information at https://www.progeriaresearch.org




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