The 10th Annual Angioma Alliance Cerebral Cavernous Malformations (CCM) Scientific Meeting

 描述（由申请人提供）：血管瘤联盟是脑海绵状血管畸形（CCM）患者的患者倡导组织，CCM是一种病因不明且无可用药物治疗的血管疾病。我们的使命是双重的：支持和教育患者社区，并推动治疗研究。CCM是一种以血管病变发展为典型特征的脑部疾病，血管病变为多海绵状薄壁结构，紧密连接不足，有出血倾向。病变出血可导致多种临床症状，包括最严重的出血性卒中和/或死亡。虽然病变发展和进展的生物学机制仍在不断阐明，但患者群体尚未获得药物治疗。唯一的治疗方法仍然是手术切除病变;然而，手术选择可能不适合所有患者，特别是那些有多发性和/或深部脑病变的患者。促进 对CCM的药物治疗的进步和快速发展的研究是CCM患者群体努力的主要动机。为了促进创新和合作研究，血管瘤联盟致力于主办年度CCM科学会议。本次会议汇集了来自研究，宣传和政府机构的利益相关者，分享和讨论未发表的研究，以推动创新并产生独特的多学科假设。世界上没有其他会议吸引了相同的研究人员-去年的会议包括外科医生和内科医生，遗传学家和微生物学家，以及专注于鼠类，斑马鱼，C。线虫和果蝇模型。这次会议的优势，以及与会者承诺每年出席的原因，是互动讨论，数据共享，假设生成和协作的机会。重要的是，会议组织者坚持介绍未发表的工作;因此，CCM科学会议通常是第一次会议的地点。 公开介绍对推进该领域至关重要的重要发现。该提案的主要目标是主办CCM十周年科学会议。该系列会议的九年历史是CCM研究领域的成功和进步之一，其中很大一部分可以归功于系列会议上发起的新合作。CCM首次会议是一个为期一天的非正式研讨会，研究人员可以面对面地讨论他们未发表的数据-议程灵活，讨论热烈。这个小型研讨会已经发展成为研究CCM研究的任何和所有方面的首要国际科学会议。2014年CCM科学会议的主要目标是：1。通过关注来自不同研究者的新研究成果，并促进研究者之间的跨学科互动，扩大并继续建立过去会议的成功; 2.通过专家小组讨论，将基础和临床前研究结果转化为临床试验工作的包容性、协作性和全球性行动计划;为初级研究者提供教育和培训机会。

 DESCRIPTION (provided by applicant): Angioma Alliance is the Patient Advocacy Group for and by those affected by Cerebral Cavernous Malformations (CCM) - a vascular disorder with unknown disease etiology and no available pharmacological treatment. Our mission is two-fold: to support and educate the patient community and to drive research for a cure. CCM is a brain disease typified by development of vascular lesions that are multi- cavernous, thin-walled structures with inadequate tight junctions and a propensity for hemorrhage. Lesion hemorrhage can result in a variety of clinical symptoms including, most severely, hemorrhagic stroke and/or death. While the biological mechanisms for lesion development and progression continue to be elucidated and no pharmacological treatment is yet available for the patient community. The only treatment remains surgical removal of the lesion; however, a surgical option may not be suitable for all patients, particularly those with multiple and/or deep brain lesions. Facilitating research for the advancement and rapid development of a pharmacological treatment for CCM is the prime motivation for the efforts of the CCM patient community. In order to facilitate novel and collaborative research, Angioma Alliance is committed to hosting the annual CCM Scientific Meeting. This meeting brings together stakeholders from research, advocacy, and government agencies to share and discuss unpublished research in an effort to drive innovation and generate unique and multidisciplinary hypotheses. No other meeting in the world draws together the same cohort of investigators - last year's meeting included surgeons and physicians, geneticists and microbiologists, as well as animal research teams focusing on murine, zebrafish, C. elegans and Drosophila models. The strength of this meeting, and the reason attendees commit to annual attendance, is the opportunity for interactive discussions, data sharing, hypothesis generation, and collaboration. Importantly, the meeting organizers insist on the presentation of unpublished work; therefore, the CCM Scientific Meeting is often the site for first public presentation of important findings that are critical for advancing the field. The primary objective of this proposal is to host the 10th anniversary CCM Scientific Meeting. The nine-year history for this meeting series is one of success and progress in the field of CCM research, much of which can be attributed to the new collaborations initiated at the meeting series. The inaugural CCM meeting was held as an informal single-day workshop for investigators to meet face-to-face and discuss their unpublished data - the agenda was flexible and the discussions were lively. This small workshop has grown to become the premier international scientific conference for those studying any and all aspects of CCM research. The primary objectives of the 2014 CCM Scientific Meeting are: 1. To expand and continue to build upon past meeting success by focusing on new research findings from diverse investigators and facilitating interdisciplinary interactions between investigators who otherwise would not have the opportunity to meet face-to-face; and 2. To translate basic and preclinical findings to develop an inclusive, collaborative and global action plan for clinical trial efforts using a moderated exper panel discussion; and 3. To provide educational and training opportunity for junior investigators.