Check out our newsletter to read about FDA approval for the first-ever treatment for Progeria, learn how the research we fund has made remarkable progress toward the cure through genetic and RNA therapies, and get the scoop on all the exciting milestones we’re celebrating right now. Here’s a sneak-peek from our two lead stories:
First-ever Treatment for Progeria Receives FDA Approval
On November 20, 2020, in partnership with Eiger Biopharmaceuticals, PRF achieved an important piece of our mission: lonafarnib, the first-ever treatment for Progeria, was granted U.S. Food & Drug Administration (FDA) approval. Progeria now joins fewer than 5% of the 7,000 known rare diseases with an FDA-approved treatment.
Breakthroughs in the Cutting-Edge Fields of Genetic and RNA Therapies
There has been tremendous progress recently in two types of therapy that could one day translate into the cure for Progeria. One study of genetic editing in a mouse model of Progeria corrected the mutation that causes Progeria in many cells, improved key disease symptoms and dramatically increased lifespan in the mice. Two other studies on the use of RNA therapeutics showed significant reduction of the toxic progerin-producing RNA, and substantial increase in survival in the mice.