Attentional Dysfunction in Children with Phenylketonuria

描述(由申请人提供)： 这项有指导的以患者为导向的研究职业发展奖将为申请人在代谢先天错误领域以患者为导向的临床研究职业生涯做好准备。代谢紊乱儿童的认知度和存活率的提高产生了对受过临床研究培训的代谢临床医生的极大需求。特别是，这些儿童的认知和行为特征在很大程度上仍未得到研究，世界范围内几乎没有临床医生拥有进行这些研究所需的技能和培训。应聘者计划：1)获得临床研究技能的教学指导，以及熟悉认知和神经心理学的基础知识；2)完成一项关于苯丙酮尿症(PKU)儿童注意力障碍的指导临床研究。这位候选人的长期目标是开发一系列有资金支持的临床研究，以改善患有苯丙酮尿症和其他先天性代谢错误的儿童的生物和心理结果。罗切斯特大学医学和牙科学院为开展临床研究提供了丰富的环境，最近开发了一套正式的课程，用于培训候选人进行临床研究。在五年期限结束时，申请者将拥有进行资助临床研究所需的技能，申请者的工作将有可能为代谢先天错误领域以患者为导向的临床研究做出重大贡献。

DESCRIPTION (provided by applicant): This Mentored Patient-Oriented Research Career Development Award will prepare the applicant for a career in patient-oriented clinical research in the field of inborn errors of metabolism. Increased recognition and survival in children with metabolic disorders have created a great need for metabolic clinicians trained in clinical research. In particular the cognitive and behavioral profiles of children these children remain largely unexplored and worldwide few clinicians have the requisite skills and training to conduct these studies. The candidate plans to: 1) acquire didactic instruction in clinical research skills as well as familiarity with the fundamentals of cognition and neuropsychology, and 2) complete a mentored clinical research study on attentional dysfunction among children with phenylketonuria (PKU). The candidate's long-term goal is to develop a series of funded clinical investigations to improve biological and psychological outcomes in children with phenylketonuria and other inborn errors of metabolism. The University of Rochester School of Medicine and Dentistry provides a rich environment for conducting clinical research, and recently developed a formal curriculum for training candidates in clinical research. At the completion of the five-year period, the applicant will have the skills necessary to conduct funded clinical research, and the applicant's work will have the potential to contribute significantly to the body of patient-oriented clinical research in the field of inborn errors of metabolism.