Our Story
In 1998, Drs. Leslie Gordon and Scott Berns received a devastating diagnosis for their son Sam: Progeria. No treatments existed. No research programs. No path forward. Only a disease that had been largely ignored for over a century.
So we built one. Leslie and Scott, together with Leslie's sister Audrey Gordon, and other family, friends, and colleagues, founded The Progeria Research Foundation, and built from scratch the entire research ecosystem the field desperately needed: a global patient registry, a cell and tissue bank, a research grants program, international scientific workshops, and a clinical trials infrastructure.. The scientific groundwork laid those early years led to identifying the cause of Progeria: a genetic mutation that produces the toxic protein progerin. We had our target.
What followed was a steady march from discovery toward treatment. In 2007, with Boston Children’s Hospital, PRF launched the first-ever Progeria clinical trial, and in 2020, the FDA approved lonafarnib as the first ever treatment for Progeria. Long-term use of this drug extends lifespan by 30%, on average!
What followed was a steady march from discovery toward treatment. In 2007, with Boston Children’s Hospital, PRF launched the first-ever Progeria clinical trial, and in 2020, the FDA approved lonafarnib as the first ever treatment for Progeria. Long-term use of this drug extends lifespan by 30%, on average!
We had built something UNIQUE: a philanthropy-funded model that drives medicine from bench to bedside for the patients no one else is coming to save.
10,000+
known rare
diseases
<5%
have an approved treatment...
<1%
...for ultrarare diseases
like Progeria
WHAT COMES NEXT
We are not done. Today, our Progeria Gene Team is advancing a gene therapy that could permanently correct the Progeria mutation. Early mouse model results are remarkable, and we are moving toward clinical trials that will determine whether this gene-editing approach is our path to the cure. This is the most ambitious chapter in our history, and it is only possible because of the model we built and the community that sustains it.
The progress is yours
Children and families who participate in research with extraordinary courage
Volunteers, staff, and board members who keep the mission moving
Scientists and clinicians who dedicate their careers to this work
Donors who generously make it possible
PRF remains the only organization in the world solely dedicated to finding treatments and the cure for Progeria.
What began as one family's refusal to accept that nothing could be done for their child has grown into a global effort connecting researchers, clinicians, and families across continents. Twenty-five years in, the work continues, and the finish line is coming into view.
