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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. Attendees included world-class physicians, scientists and preclinical investigators, along with families of children and young adults with Progeria.

Patient family presentations led the program, followed by updates on Progeria’s first-ever application for FDA drug approval (which was granted later that month), as well as initial results from the only current Progeria clinical trial. This was followed by presentations of unpublished preclinical data on drugs in development targeting the disease-causing DNA mutation, the aberrant mRNA, progerin protein, and its downstream effector proteins. Tying bench to bedside, clinicians presented new discoveries on the natural history of disease to inform future clinical trial development and new Progeria aortic valve replacement procedures. The program engaged the Progeria research community as a single unit with a common goal – to treat and cure children with Progeria worldwide.

For a full summary of the key scientific pathways being explored by top minds in Progeria research, click here to read the article.