Title : About progeria - The ultra-rare, fatal "rapid-aging" disease - And how you can help
Abstract:
Ms. Gordon will give an overview of the ultra-rare, fatal, “rapid-aging” disease, Progeria, and how those in contact with a patient with a suspected case of Progeria can help them. She will discuss signs and symptoms of Progeria, global prevalence, and The Progeria Research Foundation’s (PRF) progress that resulted in finding a treatment which gives these children longer, healthier lives, while we press onward toward the cure. Without lonafarnib (Zokinvy) treatment, children with Progeria die of heart disease at an average age of 14.5 years. PRF has many services to help these children and young adults around the world, including genetic testing to confirm diagnosis, treatment guidelines for daily care, clinical drug trials, a private families-only platform to connect with others impacted by Progeria, and programs that enable those with Progeria to access lonafarnib treatment, which has been shown to improve the cardiovasculature and extend lifespan by an average of 30% with long-term use. Over the course of PRF’s 25-year history, this rare condition has gone from obscurity to worldwide awareness. We are making extraordinary progress, from no research to a life-extending FDA- and EMA-approved treatment, development of genetic and other drug therapies that hold great promise as a future CURE, and have established healthcare guidelines - all of which give these special children longer, healthier lives, while we continue pressing forward with only the most promising research endeavors. Through our international scientific workshops we host and research grants we fund, we foster collaboration among clinicians and researchers. We have made tremendous progress toward a cure as a result of all of this activity, and invite all of you to partner with us in this quest to find, diagnose, treat, and one day cure all those around the world with Progeria.
