Highlights of the 2007 Progeria Research Foundation scientific workshop: progress in translational science.

摘要

IN the spring of 2007, a 2-year clinical drug trial began at Children's Hospital Boston (1) with the goal to advance the quality of life for children with Hutchinson-Gilford progeria syndrome (henceforth progeria), a rare (frequency 1 in 4 million), multisystem, and inevitably fatal disease that claims young lives due to myocardial infarctions and strokes between ages 7 and 20 years (2). The journey to the first clinical trial for progeria has been facilitated by a series of collaborative scientific workshops organized by The Progeria Research Foundation (PRF) and supported by agencies interested in aging, cardiovascular disease, rare disease research, and genetics (http://www.progeriaresearch.