Pediatric Dermatology Research Alliance Annual Conference

描述（由申请人提供）：儿科皮肤科医生护理患有各种罕见和常见疾病的儿童，如需要全身治疗的严重炎症性皮肤病，毁容的血管胎记和可能危及生命的遗传性皮肤病。FDA批准的治疗这些严重疾病的方法很少，并且非常需要标准化的治疗方案和方法来测量对治疗的反应。儿科皮肤病学研究联盟（Pediatric Dermatology Research Alliance，简称佩德拉）就是为了解决这一治疗不足而成立的。佩德拉的愿景是创建和维持合作研究网络，进行精心设计的高质量多中心研究，以更好地了解，预防，治疗和治愈儿童皮肤病。佩德拉年度会议是实现这些目标的核心，将包括教学讲座，由专家领导的互动讨论， 研究方法，海报会议和以疾病为重点的小组会议。具体目标是：1）为新的和成熟的佩德拉研究人员提供必要的知识基础和基础设施，以进行儿科皮肤病的高质量多中心研究; 2）在基础科学家和临床医生之间建立桥梁，以增加转化研究计划的机会; 3）确定并优先考虑四个疾病特定领域的临床需求和治疗机会：炎性皮肤病、血管瘤、大疱性表皮病和角质化病症。另一个目标是将研究使命扩大到现有工作组以外，包括其他有需要的疾病。

DESCRIPTION (provided by applicant): Pediatric dermatologists care for children with a broad range of rare and common disorders, such as severe inflammatory skin diseases that require systemic therapy, disfiguring vascular birthmarks, and genetic skin disorders that can be life-endangering. There is a paucity of FDA-approved therapies for these severe conditions and a great need for standardized treatment protocols and methods to measure response to therapy. The Pediatric Dermatology Research Alliance (PeDRA) was established to address this therapeutic shortfall. PeDRA's vision is to create and sustain collaborative research networks that conduct well-designed, high-quality multicenter studies to better understand, prevent, treat and cure dermatologic diseases in children. The PeDRA Annual Conference is central to achieving these goals and will include didactic lectures, interactive discussions led by experts in research methodology, poster sessions, and disease-focused small-group meetings. The specific aims are: 1) To provide new and established PeDRA investigators with an essential knowledge base and infrastructure to conduct high-quality multicenter research in pediatric skin disorders; 2) To create a bridge between basic scientists and clinicians to enhance opportunities for translational research initiatives; and 3) To identify and prioritize the clinical needs and therapeutic opportunities in four disease- specific areas: inflammatory skin diseases, hemangiomas, epidermolysis bullosa, and disorders of keratinization. An additional goal is to expand the research mission beyond the established working groups to other disorders with need.