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We’re thrilled to share the results from two very exciting breakthrough studies on the use of RNA therapeutics in Progeria research. Both studies were co-funded by The Progeria Research Foundation (PRF) and co-authored by PRF’s Medical Director, Dr. Leslie Gordon.

In January, the science journal Природа published breakthrough results demonstrating that genetic editing in a mouse model of Progeria corrected the mutation that causes Progeria in many cells, improved several key disease symptoms and dramatically increased lifespan in the mice.

We’re thrilled to announce that today, we achieved an important piece of PRF’s mission: lonafarnib, the first-ever treatment for Progeria, has been granted FDA approval.

Read about how our progress continues despite the pandemic; the science driving PRF’s latest grant recipients to propel Progeria research to new heights; inspiring thoughts from families in our Progeria community; and so much more.

From all of us at PRF, as well as the children and their families, THANK YOU to everyone who donated to our ONEpossible campaign!!
We CRUSHED our goal – which will help to offset funds lost from cancelled events due to COVID-19 – and couldn’t have done it without your support!

First and foremost, we hope you are staying well. In light of the recent progression of COVID-19, and as we all navigate this uncertain time, we want to THANK YOU for your support and let you know that our fight against Progeria remains steadfast.