Sixth International Myotonic Dystrophy Consortium Meeting

描述（由申请人提供）：第六届国际强直性肌营养不良联盟（IDMC）将汇集整个研究人员，临床医生，其他护理提供者，患者和家庭成员，讨论当前的研究发现，新的治疗方法和强直性肌营养不良的最佳临床护理。除了关注这些领域的最新科学进展外，会议的目标还包括：1）加强强直性肌营养不良基础和临床研究相关信息的公开交流; 2）促进全球研究者之间的合作研究，提高对基本病理机制的理解，制定最佳的转化研究策略，并确定新疗法的优先领域; 3）为初级研究人员提供展示其工作并与该领域更成熟的研究人员和临床医生互动的机会，并鼓励患者及其倡导者与这些年轻和高级研究人员会面，讨论他们对强直性肌营养不良的共同兴趣和担忧。本次会议在分享新信息、促进科学交流和形成合作方面有着良好的记录，并提供了一个独特的环境，将研究人员、临床医生和患者整合在一起，就研究的重要性进行深入的对话，特别是在涉及患者和未来治疗的情况下。这次会议是鼓励培训青年科学家（研究生、博士后研究员和PI）的绝佳场所。IDMC是对该领域前沿进展进行全面、广泛讨论的首要会议。基因缺陷的发现和负责特定疾病表现的分子病理机制的鉴定为开发针对这一无情地进行性疾病组的有效疗法创造了新的机会。

DESCRIPTION (provided by applicant): The Sixth International Myotonic Dystrophy Consortium (IDMC) will bring together the entire spectrum of researchers, clinicians, other care providers, patients and family members to discuss current research discoveries, new therapeutic approaches, and optimal clinical care for myotonic dystrophy. In addition to focusing upon the most recent scientific advances in these areas, the goals of the meeting include: 1) To enhance the open exchange of the information related to basic and clinical research in myotonic dystrophy; 2) To stimulate the initiation of collaborative research between investigators worldwide, to improve the understanding of the fundamental pathomechanism, to develop optimal translational research strategies, and to identify high priority areas for new therapies; 3) To provide junior investigators with an opportunity to present their work and to interact with more established researchers and clinicians in the field, and to encourage patients and their advocates to meet with these young and senior investigators to discuss their common interests and concerns about myotonic dystrophy. This meeting has an established record of sharing new information, stimulating scientific exchange, and forming collaborations, as well as providing a unique environment that integrates researchers, clinicians, and patients in an extended conversation about the significance of research especially as it pertains to patients and future treatments. This meeting is an excellent venue for encouraging the training of young scientists (graduate students, post-doctoral fellows and PIs). The IDMC is the premier meeting for a comprehensive, broad-based discussion of cutting edge advances in the field. Discoveries of the gene defects and identification of molecular pathomechanisms responsible for specific disease manifestations have created new opportunities to develop effective therapies for this relentlessly progressive group of diseases.