ESTABLISH THE NATIONAL EPIDERMOLYSIS BULLOSA REGISTRY

点击翻译按钮获取中文摘要

The purpose of this contract is to establish a National Epidermolysis Bullosa Registry aimed at searching for the basic defect, improving methods of diagnosis, and developing effective methods in treatment and prevention. The Contractor will establish and operate a Clinical Coordinating Center under the Registry. The Registry will (1) develop a roster of well characterized patients with different forms of epidermolysis bullosa; (2) determine with accuracy and precision the incidence and prevalence of disease; (3) assess disease distribution geographically to search for clusters and patterns, and assess the societal and economic impact of the disease; (4) develop a pool of patients of the same genetic type for a wide variety of studies on pathogenesis and genetics of E.B.; and (5) foster well designed and well executed clinical trials of new therapeutic interventions in carefully selected groups of patients with one or another form of the disease.