Funding for FightSMA Researchers' Conference in Washington, DC, April 2008

为 2008 年 4 月在华盛顿特区举行的 FightSMA 研究人员会议提供资金

• 批准号: 7487724
• 负责人: Christian L. Lorson
• 金额: $ 3.3万
• 依托单位: ANDREW'S BUDDIES CORPORATION
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-04-15 至 2009-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7487724
• 关键词: Address; Amyotrophic Lateral Sclerosis; Cardiac; Categories; Child; Chronic; Clinical; Clinical Trials; Collaborations; Communication; Consultations; Country; Development; Discipline; Disease; District of Columbia; Doctor of Philosophy; Duchenne muscular dystrophy; Face; Family; Feedback; Friends; Funding; Gel; Genetic; Goals; Health Professional; Hereditary Disease; Individual; Infant; Institutes; Institution; International; Interview; Lung; Massachusetts; Medical; Medical center; Missouri; Muscular Dystrophies; Newly Diagnosed; Numbers; Occupational Therapy; Ohio; Ontario; Parkinson Disease; Patients; Pediatric Hospitals; Public Health; Research; Research Personnel; Science; Specialist; Spinal Muscular Atrophy; Stroke; Technology; Testing; Thinking; Translational Research; Tube; Universities; Update; Utah; Vision; Work; day; design; experience; fight against; fighting; innovation; insight; medical schools; nervous system disorder; novel strategies; programs; respiratory; symposium; therapy development

DESCRIPTION (provided by applicant): The FightSMA Researchers' Conference is designed as a forum where innovative scientific approaches and new research findings can be presented and reviewed. This information is meant to promote the continued quest for a cure for SMA and related disorders. Spinal Muscular Atrophy is the number one genetic-related killer of infants in the US. Research related to SMA has been proven relevant to at least forty other diseases. It is imperative that this year's program on April 20th-22nd, 2008, continue to bring the best and the brightest together to develop treatment for SMA and related disorders. Each year Andrew's Buddies dba FightSMA hosts a conference in Washington, DC to: Bring specialists in the fields of Spinal Muscular Atrophy (SMA) and related neurological diseases together to break down the academic and disease-specific silos and integrate different domains and disciplines in developing a cure for SMA. The networking and collaboration among investigators also encourages the development of therapies that not only would benefit SMA, but other related illnesses such as ALS. Facilitate communication between SMA researchers and SMA families. Families of SMA patients arrive the last day of the Researchers' Conference for a Family and Friends Conference. This Conference utilizes the information and expertise of the SMA investigators to familiarize families with SMA research and clinical trials. Several researchers participate in panelist discussions and one-on-one interviews with SMA families to discuss patient-specific concerns. This interaction between patient and professional not only provides families with valuable insight, but also allows the specialists to meet with the individuals who benefit from their research. Without fail, this is one of the most important aspects of the annual meeting. Patients are able to talk one- on-one with clinicians and researchers, but equally importantly, researchers are able to put a face and connect with the patients on a personal level. This type of interaction truly drives investigators and emphasizes the importance of the day-to-day research when test tubes and gels can seem mundane and removed from the ultimate goal. Andrew's Buddies dba FightSMA recognizes the importance of bringing together national and international researchers to discuss their results and conclusions. This think tank of medical professionals includes researchers who have been involved in fighting SMA for the past 20+ years, as well as younger researchers who are investigating new cutting edge technologies for finding a cure. Worldwide the number of SMA researchers has grown dramatically over the past decade, however, during this meeting, attendance is intentionally restricted to allow for a greater level of discussion and openness that larger venues usually can offer. The Conference agenda includes individual presentations and panelist discussions in three distinct categories: Clinical, Scientific and Translational Research. Investigators relay what they have discussed during the two and a half day conference to their institutions and their colleagues and, when applicable, apply these strategies to their research. PUBLIC HEALTH RELEVANCE: This two and a half day conference brings together over twenty investigators in the field of Spinal Muscular Atrophy (SMA) and related chronic neurological diseases such as ALS and Parkinson's. These researchers share their findings and work together to develop a practical approach to developing not only a cure for SMA, but also investigative therapies to benefit other illnesses. A lay-friendly science briefing, panelist discussions, and one-on-one consultations are conducted by a select group of SMA researchers and the FightSMA Science Director at the Friends and Family Conference immediately following the Researchers' Conference.

描述（由申请人提供）：FightSMA 研究人员会议被设计为一个可以展示和审查创新科学方法和新研究成果的论坛。该信息旨在促进对 SMA 及相关疾病治愈方法的持续探索。脊髓性肌萎缩症是美国婴儿的第一大遗传相关杀手。与 SMA 相关的研究已被证明与至少 40 种其他疾病相关。今年的计划（2008 年 4 月 20 日至 22 日）必须继续汇集最优秀和最聪明的人才来开发 SMA 和相关疾病的治疗方法。 Andrew's Buddies dba FightSMA 每年都会在华盛顿特区举办一次会议，目的是： 将脊髓性肌肉萎缩症 (SMA) 和相关神经系统疾病领域的专家聚集在一起，打破学术和特定疾病的孤岛，并整合不同领域和学科来开发SMA 的治疗方法。研究人员之间的网络和合作也鼓励了疗法的开发，这些疗法不仅有益于 SMA，而且有益于 ALS 等其他相关疾病。促进 SMA 研究人员和 SMA 家庭之间的沟通。 SMA 患者家属在家庭和朋友会议研究人员会议的最后一天抵达。本次会议利用 SMA 研究人员的信息和专业知识，让家庭熟悉 SMA 研究和临床试验。几位研究人员参与小组讨论和与 SMA 家庭的一对一访谈，讨论患者的具体问题。患者和专业人士之间的这种互动不仅为家属提供了宝贵的见解，而且还使专家能够与从他们的研究中受益的个人会面。毫无疑问，这是年会最重要的方面之一。患者能够与临床医生和研究人员进行一对一的交谈，但同样重要的是，研究人员能够在个人层面上与患者进行面对面的交流。当试管和凝胶看起来很平凡并且远离最终目标时，这种类型的互动真正推动了研究人员并强调了日常研究的重要性。 Andrew's Buddies dba FightSMA 认识到将国内和国际研究人员聚集在一起讨论他们的结果和结论的重要性。这个由医学专业人士组成的智库包括过去 20 多年来一直致力于对抗 SMA 的研究人员，以及正在研究新的尖端技术以寻找治疗方法的年轻研究人员。在过去的十年里，世界范围内 SMA 研究人员的数量急剧增长，然而，在本次会议期间，有意限制出席人数，以便更大的场地通常可以提供更高水平的讨论和开放性。会议议程包括三个不同类别的个人演讲和小组讨论：临床、科学和转化研究。研究人员将他们在为期两天半的会议中讨论的内容转达给他们的机构和同事，并在适用的情况下将这些策略应用到他们的研究中。公共健康相关性：这次为期两天半的会议汇集了脊髓性肌萎缩症 (SMA) 和相关慢性神经系统疾病（如 ALS 和帕金森病）领域的 20 多名研究人员。这些研究人员分享他们的发现，并共同开发一种实用的方法，不仅可以治愈 SMA，还可以开发有益于其他疾病的研究疗法。在研究人员会议结束后，由 SMA 研究人员和 FightSMA 科学总监组成的精选小组在朋友和家人会议上进行了一场非专业人士友好的科学简报、小组讨论和一对一咨询。

项目成果

Cor Triatriatum Sinistrum: Classification and Imaging Modalities.

Cor Triatriatum Sinistrum：分类和成像方式。

• 发表时间: 2011-01
• 作者: Nassar, Pierre Nagib;Hamdan, Righab Haidar
• 通讯作者: Hamdan, Righab Haidar