Midcareer Investigator Award in Patient-Oriented Research for Dr. Ryan Branski

项目摘要/摘要 地震影响了全球约1%的人口，即近8000万人。尽管有这个惊人的数字，但两者 命名和治疗方法不一致。许多患者出现震颤，影响到 发声，单独发声或合并四肢发抖。孤立性发声震颤在历史上是 被认为是原发性震颤(ET)的临床变种。然而，最近更新的分类标准不包括 从ET分类中分离出声音颤动。这场争论为有意义的患者提供了一个理想的平台- 面向研究，有可能实质性地解决与沟通相关的残疾问题。 此外，这个K24职业中期奖将使我能够提高我提供的指导的数量和质量 给学生、住院医生、研究员和初级教员。我这次获奖的研究重点将是理解 这两种患者的临床表型差异以及声音震颤对其的影响 上呼吸道结构和吞咽。此外，关于以下方面的基础疗效数据 将收集针对声音颤抖患者的药物治疗。 职业目标和研究目标，与国家聋人研究所的使命和 其他沟通障碍，被推荐为这个K24职业生涯中期奖。关于职业生涯 在发展和指导方面，提出了以下职业目标： 职业目标1.评估我的导师的质量，并通过 正式的培训计划 职业目标2.指导三年级学生、博士后学者、住院医生、研究员和教职员工翻译 科学通过科学发现加强对患者的护理 此外，还组建了一个多学科小组，以实现以下研究目标： 目的1.研究ETVT和IVT患者重叠和/或区分的临床表型。 目的2.研究室性心动过速严重程度与ETVT和IVT患者吞咽改变的关系。 目标3(探索性)。全身用药与局部用药治疗ETVT的疗效比较 和IVT。

PROJECT SUMMARY/ABSTRACT Tremor affects ~1% of the global population or nearly 80 million people. In spite of this staggering figure, both nomenclature and treatment approaches are inconsistent. Many patients present with tremor affecting the voice, either in isolation or in combination with tremor of the limbs. Isolated vocal tremor was historically considered a clinical variant of essential tremor (ET). Recently updated classification criteria, however, exclude isolated voice tremor from ET classification. This controversy provides an ideal platform for meaningful patient- oriented research with the potential to substantively address issues related to communication-related disability. In addition, this K24 midcareer award will allow me to enhance the quantity and quality of mentoring I provide to students, residents, fellows, and junior faculty. My research focus for this award will be on understanding differences in the clinical phenotypes between these two patient populations and the impact of voice tremor on structures of the upper aerodigestive tract and deglutition. In addition, foundational efficacy data regarding pharmacological therapy for patients with voice tremor will be collected. Both Career Goals and Research Aims, consistent with the mission of the National Institute on Deafness and Other Communication Disorders, are proposed for this K24 midcareer award. With regard to career development and mentorship, the following Career Goals are proposed: Career Goal 1. To evaluate the quality of my mentoring and implement a plan for improvement through a formalized training plan Career Goal 2. To mentor junior students, post-doctoral scholars, residents, fellows, and faculty in translational science to enhance patient care through scientific discovery In addition, a multi-disciplinary team has been assembled to address the following Research Aims: Aim 1. To characterize overlapping and/or distinguishing clinical phenotypes of patients with ETVT and iVT. Aim 2. To correlate VT severity with altered deglutition in ETVT and iVT. Aim 3 (Exploratory). To compare systemic and locally administered pharmacological treatment effects for ETVT and iVT.