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

描述（由申请人提供）：搏击sma研究人员会议旨在作为一个论坛，创新的科学方法和新的研究成果可以展示和审查。这一信息旨在促进对SMA和相关疾病治疗的持续探索。脊髓性肌萎缩症是美国婴儿的头号基因相关杀手。与SMA相关的研究已被证明与至少40种其他疾病有关。2008年4月20日至22日的今年的项目，必须继续把最优秀和最聪明的人聚集在一起，共同开发SMA和相关疾病的治疗方法。每年，Andrew's Buddies dba FightSMA都会在华盛顿特区举办一次会议，将脊髓性肌萎缩症（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壁垒，整合不同领域和学科，共同开发治疗SMA的方法。研究人员之间的网络和合作也鼓励了治疗方法的发展，不仅有利于SMA，也有利于其他相关疾病，如ALS。促进SMA研究人员和SMA家庭之间的沟通。肌萎缩萎缩症患者的家属到达研究人员会议的最后一天，参加一个家庭和朋友会议。本次会议利用SMA研究人员的信息和专业知识，使家庭熟悉SMA研究和临床试验。几位研究人员参加了小组讨论和与SMA家庭的一对一访谈，以讨论患者的具体问题。患者和专业人员之间的互动不仅为家庭提供了宝贵的见解，而且还允许专家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。患者可以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，研究人员可以在个人层面上与患者进行面对面的交流。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，当试管和凝胶看起来平凡而远离最终目标时。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囊团包括过去20多年来一直参与对抗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，全球SMA研究人员的数量急剧增长，然而，在这次会议期间，出席人数被有意限制，以允许更大程度的讨论和开放，而这通常是大型会场所能提供的。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。研究人员将他们在两天半的会议期间讨论的内容传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于他们的研究。公共卫生相关性：这个为期两天半的会议汇集了20多位脊髓性肌萎缩症（SMA）和相关慢性神经系统疾病（如ALS和帕金森病）领域的研究人员。这些研究人员分享了他们的发现，并共同开发了一种实用的方法，不仅可以治疗SMA，还可以研究治疗其他疾病的方法。在研究人员会议之后的朋友和家庭会议上，由一组SMA研究人员和FightSMA科学主任进行了一次外行友好的科学简报、小组讨论和一对一的咨询。

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.