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

项目总结/摘要 震颤影响约1%的全球人口或近8000万人。尽管这一数字令人震惊， 命名和治疗方法不一致。许多患者出现震颤， 声音，无论是在孤立或与四肢震颤相结合。孤立性声带震颤在历史上 被认为是特发性震颤（ET）的临床变体。然而，最近更新的分类标准排除了 孤立的声音震颤从ET分类。这场争论为有意义的病人提供了一个理想的平台- 有可能实质性地解决与沟通有关的残疾问题的研究。 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 学生、住院医生、研究员和初级教员。我对这个奖项的研究重点将是理解 这两个患者群体之间临床表型的差异以及声音震颤对 上呼吸消化道的结构和吞咽。此外，关于以下方面的基础疗效数据 将收集声音震颤患者的药物治疗。 职业目标和研究目标，符合国家聋人研究所的使命， 其他沟通障碍，建议为这个K24职业中期奖。关于职业 发展和指导，提出了以下职业目标： 职业目标1.评估我的指导质量，并通过 正规化训练计划 职业目标2.指导大三学生，博士后学者，居民，研究员和教师在翻译 通过科学发现提高病人护理水平 此外，还组建了一个多学科团队，以实现以下研究目标： 目标1.表征ETVT和iVT患者的重叠和/或区别临床表型。 目标二。将VT严重程度与ETVT和iVT的吞咽改变相关联。 目标3（探索性）。比较全身和局部给药对ETVT的药理学治疗效果 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.