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

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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.