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About

Mission

To discover treatments and the cure for Progeria and its aging-related disorders, including heart disease.

Vision

A world in which every child with Progeria is cured.

Values

PRF is a vibrant organization with a deep commitment to finding treatments and the cure for Progeria. We operate in a fast-paced, dynamic environment that values innovation, collaboration, and integrity.  Our values define who we are, guide expected behaviors at all levels of the organization, and provide the framework for how we interact with those with progeria and their families, progeria researchers and clinicians, research-related program partners, donors, volunteers, and other supporters of our mission:

  • PRF staff and Board are committed to and passionate about the work we do each day.
  • Our programs are innovative and research-driven. We are leaders in our field.
  • Business operations are transparent while respectful of the privacy of our donors and the families we serve.
  • We sustain and reinvest in our mission by wisely managing our human and material resources, recognizing the responsibility and accountability we have for stewardship of the resources entrusted to us.
  • We strive to provide information that is accurate, objective, relevant, timely and understandable.
  • Teamwork is an integral part of everything we do, as we truly believe that Together, we WILL find the cure!

Our Story

Progeria is a rare, fatal, “rapid aging” disease. Without lonafarnib treatment, all children with Progeria die of heart disease at an average age of 14.5 years. The Progeria Research Foundation (PRF) was founded in 1999 in response to the complete lack of progress being made to help children with Progeria.

Today, PRF continues to be the only organization in the world solely dedicated to finding treatments and the cure for Progeria. We have filled a void, taking these children out of the background where they had been for over 100 years and putting them and Progeria at the forefront of scientific efforts.

In just a relatively short time, we have achieved extraordinary progress towards our mission: the Progeria gene discovery in 2003, first-ever clinical drug trials initiated in 2007 to explore a drug called lonafarnib, and approval by the U.S. Food and Drug Administration (FDA) in 2020 for lonafarnib, the first-ever treatment for Progeria, which is now the standard-of-care. This historic milestone put Progeria among the 5% of rare diseases that have an FDA approved treatment! In addition, we’ve achieved extensive global awareness of the disease and PRF’s work, and confirmation of critical biological links between Progeria, heart disease and the aging we all experience. In 2023, PRF researchers developed a biomarker for Progeria, enabling researchers to more quickly and accurately gauge the effectiveness of new treatment candidates much earlier in a clinical trial, accelerating the pace of progress toward better treatments and the cure.

From an obscure, ignored disease at our founding in 1999, to treatment and global recognition today – we celebrate an unprecedented timeline of progress in the world of medical research! Our accomplishments are being praised as a “scientific sprint” as we forge ahead in this race against time.

All of this progress is due in large part to the establishment of PRF’s research-related programs and services. Developed with insightful determination, they provide the resources needed not only to advance the field of Progeria, but also to discover what Progeria can tell us about heart disease and aging.

With the support of dedicated staff and volunteers, a talented board of directors, courageous families, and thousands of generous people around the world, we are pushing Progeria research forward towards discovery, treatments, and cure. And along the way, we are learning a tremendous amount about ourselves.

Please enjoy the pages of this website, which detail PRF’s programs, progress and partners. The historic Progeria treatment discovery, tremendous success of our global campaign to identify all children with Progeria, and clinical drug trials and drug discovery are contributing to exciting and productive times as we edge ever-closer to our ultimate goal of a cure.  Your love and support for children with Progeria makes these exciting strides possible.

Together, we WILL find the cure!

Your donation helps The Progeria Research Foundation treat children with Progeria today and cure them in the future.

Progeria is a rare, fatal, “rapid aging” disease. Without the discovery of new treatments, all children with Progeria will die of heart disease at an average age of 14 years. The Progeria Research Foundation (PRF) was founded in 1999 in response to the complete lack of progress being made to help children with Progeria. Our original mission: to discover the cause, treatments and cure for Progeria.* Today, PRF continues to be the only organization in the world solely dedicated to this mission. We have filled a void, taking these children out of the background where they had been for over 100 years and putting them and Progeria at the forefront of scientific efforts.

In just a relatively short time, we have achieved extraordinary progress towards our mission: the Progeria gene discovery in 2003, first-ever clinical drug trials initiated in 2007, and the 2012 results of that first trial being the discovery of the first treatment for Progeria. In addition, we have achieved extensive global awareness of the disease and PRF’s work, and confirmation of critical biological links between Progeria, heart disease and the aging we all experience. From an obscure, ignored disease to treatment and global recognition – an unheard of timeline in the world of medical research! Our accomplishments are being praised as a “scientific sprint”** as we forge ahead in this race against time.

All of this progress is due in large part to the creation of PRF’s research-related programs and services. Developed with insightful determination, they provide the resources needed not only to advance the field of Progeria, but also to discover what Progeria can tell us about heart disease and aging.

With the support of dedicated staff and volunteers, a talented board of directors, courageous families, and thousands of generous people around the world, we are pushing the field of Progeria forward towards discovery, treatments, and cure. And along the way, we are learning a tremendous amount about ourselves.

Please enjoy the pages of this web site, which detail PRF’s programs, progress and partners. The historic Progeria treatment discovery, tremendous success of our global campaign to identify all children with Progeria, and clinical drug trials and drug discovery are contributing to exciting and productive times as we edge ever-closer to our ultimate goal of a cure.Your love and support for children with Progeria makes these exciting strides possible.

Together, we WILL find the cure!

Your donation helps The Progeria Research Foundation treat children with Progeria today and cure them in the future.