Today, the science journal Nature 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.
Enjoy some clips, photos, comments, and Q&A!
Join us on Dec. 5, 2020 for another epic Night of Wonder, this year from the comfort of your own home!
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.
During an otherwise difficult time for our world, we are happy to share a bright spot: Eiger BioPharmaceuticals has completed submission of a New Drug Application (NDA), seeking approval – in Europe and the US – of the drug lonafarnib as a first-ever treatment for Progeria.
On March 1st, PRF Ambassador Meghan Waldron turned 19, and we celebrated with March Madness 2020: Where in the world is Meghan Waldron? We've shared photos from Meghan’s travels and hope they've inspired you to think about where your next adventure will be once we're...
We’re thrilled to announce a milestone in our mission to treat and cure children with Progeria: In partnership with The Progeria Research Foundation, Eiger BioPharmaceuticals has submitted to the FDA the first part of its application seeking approval of the drug lonafarnib as a treatment for Progeria.