In November, 2020, PRF brought over 370 registrants from 30 countries ‘together’ in our first-ever virtual scientific workshop. Attendees were given a platform to share their expertise in Progeria research and meet some of the children who will benefit from their work. A summary of the workshop was published today in the journal Aging.
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 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.