The Progeria Research Foundation (PRF) is focused on finding a treatment for progeria, a disease caused by a mutation in the LMNA gene that results in premature aging. Children with Progeria begin to display many characteristics of accelerated aging at around 18-24 months of age. Progeria signs include growth failure, loss of body fat and hair, aged-looking skin, stiffness of joints, hip dislocation, generalized atherosclerosis, cardiovascular (heart) disease and stroke. Almost all children with progeria die of atherosclerosis (heart disease) at an average age of fourteen years.
Currently there is no approved treatment for Progeria. However, since 2007, PRF has spearheaded several clinical trials, including the most recent 'triple trial' involving Lonafarnib, Zoledronic acid, and Pravastatin.
In this exclusive interview with Rare Disease Report, PRF president Audrey Gordon discusses the clinical trials PRF is involved with and the role PRF is playing in getting as many progeria patients as possible on a treatment.