The 8th Annual Cerebral Cavernous Malformations (CCM) Scientific Meeting

描述（由申请人提供）：脑海绵状血管瘤（CCM）是一种中枢神经系统血管性疾病，其特征是大脑和脊髓中存在血管病变。与CCM相关的临床症状多种多样，可能包括复发性头痛、局灶性神经功能缺损、癫痫发作、出血性中风和/或死亡。CCM患者没有可用的药物治疗-通过观察或脑外科手术进行保守管理是目前唯一的治疗选择。因此，迫切需要更充分地阐明与CCM相关的病理生物学和疾病机制，以便开发预防性药物治疗。开发治疗方法并将研究成果转化为人类表型将需要研究、临床护理、宣传、行业和政府机构等利益攸关方的全球合作。促进这些进步和合作是血管瘤联盟的优先事项，血管瘤联盟是为CCM患者提供支持的组织。本提案的主要目标是主办第八届CCM年度科学会议。一年一度的血管瘤联盟CCM科学会议是国际研究人员和临床医生聚集和分享他们最新的CCM研究数据和临床发现的唯一场所。没有其他以疾病为基础的会议、研讨会或会议专门讨论脑海绵状血管瘤的主题。没有其他国家或国际会议吸引来自遗传学、血管生物学、药理学、神经病学、放射学、神经外科、蛋白质组学和细胞生物学等领域的科学家；他们都致力于充分阐明CCM的病理生物学，并将这些发现转化为人类的情况。CCM科学会议关注患者群体的未来，既寻求治疗，也促进下一代CCM科学家和医生的培训和发展。CCM研究领域持续快速发展。为了跟上这一步伐，本提案旨在申请部分资金，以帮助支持CCM科学会议计划的持续扩展。我们打算举办一次科学会议，具体目的如下：汇集关注CCM研究及相关领域的临床和基础科学研究人员。2. 为年轻的调查人员提供培训和展示机会。3. 为临床治疗的设计和实施制定行动计划

DESCRIPTION (provided by applicant): Cerebral cavernous malformations (CCM) is a vascular disease of the central nervous system characterized by the presence of vascular lesions in the brain and spinal cord. Clinical symptoms associated with CCM are variable and may include recurrent headaches, focal neurological deficits, seizures, hemorrhagic stroke and/or death. There is no pharmacological treatment available for CCM patients - conservative management by observation or brain surgery are the only current treatment options. Thus, there is a critical need to more fully elucidate the pathobiology and disease mechanisms associated with CCM so that preventative pharmacological treatments can be developed. Developing treatments and translating research findings to the human phenotype will require global collaboration from stakeholders in research, clinical care, advocacy, industry, and government agencies. Facilitating these advancements and collaborations is a priority of Angioma Alliance - the patient advocacy organization for those affected by CCM. The primary objective of this proposal is to host the 8th annual CCM Scientific Meeting. The annual Angioma Alliance CCM Scientific Meeting is the only venue for international researchers and clinicians to assemble and share their latest CCM research data and clinical findings. There are no other disease-based meetings, workshops or conferences dedicated to the topic of cerebral cavernous malformations. No other national or international meetings attract the same cohort of scientists from fields including genetics, vascular biology, pharmacology, neurology, radiology, neurosurgery, proteomics, and cell biology; all of whom are dedicated to fully elucidating the pathobiology of CCM and translating those findings to the human condition. The CCM Scientific Meeting focuses on the future for the patient community, both for seeking a cure and also by fostering the training and development of the next generation of CCM scientists and physicians. The field of CCM research continues to advance rapidly. In keeping with this pace, this proposal is designed to request partial funding to help support the continued expansion of the CCM Scientific Meeting program. We intend to host a scientific meeting with the following specific aims: 1. To gather a diverse group of clinical and basic science investigators focusing on CCM research and related fields. 2. To provide a training opportunity and showcase for young investigators. 3. To create an action plan for the design and implementation of clinical treatment trials. ! PUBLIC HEALTH RELEVANCE: Cerebral cavernous malformations (CCM) is a debilitating disease of the brain and spinal cord that can cause affected individuals to suffer recurrent headaches, hearing and vision problems, seizures and/or stroke. At this time there is no available drug treatment; the only option for most patients is to undergo brain surgery. The CCM Scientific Meeting is designed to gather the world's experts on this illness to discuss the latest n research advances and plan for future research collaborations and clinical drug trials.