The Beginning
No awareness. No research. No treatments. No organization fighting for these children. So we built one.
Our Impact
A Quarter Century of Breakthroughs
From Nothing to a Path to the Cure
1999
2003
The Gene is Found
The doors of science fly open. PRF’s Genetics Consortium identifies progerin, the toxic protein behind Progeria. For the first time, researchers have a target.
2007
The First Clinical Trial
A promising drug, lonafarnib, is identified as a potential treatment. The first-ever Progeria clinical trial begins.
2009
“Find The Other 150” Campaign
PRF’s global campaign locates children with Progeria worldwide.
The Triple Drug Trial
PRF funds a triple combination drug trial with lonafarnib + pravastatin + zoledronate. Although the trial finds no significant improvement over lonafarnib alone, the results inform future research directions.
2010
A Window Into All of Aging
Scientists discover that Progeria is linked to heart disease and normal aging. A cure for Progeria could one day help millions.
2012
The First Real Results
Trial data confirms it: lonafarnib improves the cardiovascular health and other disease symptoms.
2013
The World Meets Sam
HBO’s Life According to Sam and Sam’s TEDx talk brings Progeria into living rooms worldwide and inspires millions.
2016
Doubling Down
PRF funds a combination drug trial with lonafarnib + everolimus. Although the trial finds no significant improvement over lonafarnib alone, the results inform future research directions.
2018
Longer Lives, Proven
JAMA publishes a landmark finding: lonafarnib extends survival by 20%. The path to FDA approval is now in sight.
2020
FDA Approval
History is made. Lonafarnib (now branded as ‘Zokinvy’) becomes the first-ever FDA-approved treatment for Progeria, placing it among fewer than 5% of rare diseases with an approved drug.
2021
Gene Therapy Breakthrough
Nature publishes a landmark study that shows gene editing corrects the Progeria mutation in mice and significantly increases their lifespan
2022
Europe Says Yes
Lonafarnib is approved across Europe, bringing treatment to children and young adults on a second continent.
2023
Even Longer Lives
Updated data shows long-term lonafarnib use extends lives by an average of 30%.
Biomarker Discovery
PRF develops a new DNA test to accelerate future discoveries.
2024
Japan Says Yes
Lonafarnib is approved in Japan. The treatment is now reaching children across three continents.
2025
A Path to the Cure: PRF Becomes a Drug Developer
After 7 years of intense work, PRF and the Progeria Gene Team advance SamPro-2, a gene editing therapy designed to permanently correct the single DNA typo that causes Progeria, into the manufacturing stage.
2026
The Era of Gene Therapy
SamPro-2 moves closer to clinical trials with the announcement of a manufacturing partnership with Forge Biologics.
