The Progeria Research Foundation

What The Progeria Research Foundation makes

Topics progeria, genetics, rare-diseases, healthcare, research

The Progeria Research Foundation (PRF) is the driving force behind the global effort to understand, treat and ultimately cure children & young adults with Hutchinson-Gilford Progeria Syndrome (Progeria), a rare & fatal genetic disease that causes rapid aging in children. Founded by the family of Sam Berns after his diagnosis in 1999, PRF has enabled or led major scientific breakthroughs in the field; from discovery of the causative gene mutation, to Progeria’s first and only FDA-approved treatment, lonafarnib, to the advancement of gene-editing approaches now in development. Through rigorous science, global research infrastructure and close partnership with the worldwide patient community, PRF is advancing next-generation therapies & leading the Path to Cure Progeria program to determine whether a one-time gene-editing therapy can offer a durable, potentially curative treatment. For more information and to support PRF’s mission, please visit www.progeriaresearch.org.

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