Spinal Muscular Atrophy (SMA) is an inherited neuromuscular disease that leads to motor dysfunction and death. It affects one of 6000 babies born and is the leading genetic killer of children under two years of age. The molecular basis of the disease is a deficiency in production of a specific protein - Survival of Motor Neuron (SMN) protein. Motor neuron function is acutely sensitive to lowered SMN protein levels. This cellular defect is the underlying basis for the loss of control of muscles in the limbs, neck, and chest in these patients. For the past 12 years, Families of Spinal Muscular Atrophy (FSMA) has sponsored the Annual International Spinal Muscular Atrophy Group Meeting, the only open conference entirely dedicated to SMA research. In 2009 the conference will be held June 18th to 20th in the Hyatt Regency Cincinnati in Cincinnati, Ohio. Since its inception, the meeting has grown from 40 attendees to almost 250 in 2008. It is currently the largest research conference for SMA worldwide. The conference is held simultaneously with the FSMA Families and Professional Conference, providing researchers a unique opportunity to interact with the SMA patients they are dedicated to helping. The overall meeting goal is to bring together the leading SMA research experts from around the world to share unpublished data and foster a spirit of communication and collaboration. This is achieved in several ways. First, the conference is organized into nine distinct sessions around the major unanswered research topics in SMA research. Second, because this conference is the only open conference dedicated to SMA, it is the one venue where the entire SMA community can share new data and information on an annual basis. A secondary goal is to bring young and new researchers into the SMA community, including those located internationally, who do not have opportunities to interact with the recognized thought leaders in this area. This is the only SMA focused conference that encourages attendance and presentation by post-doctoral fellows and graduate students, rather than only having PI participation. This is an essential aspect of the meeting as it helps build the future of the SMA research community.
Spinal Muscular Atrophy is a neuromuscular disease affecting 1 in 6000 live births. It is the leading genetic killer of children under 2 years of age. SMA results in a large unmet medical need throughout the world, and researchers have suggested that SMA is one of the neurological diseases closest to treatment. SMA is caused by mutations of the SMN1 gene, which is deleted in over 90% of patients. Due to the unique presence of a back-up gene for SMN1 called SMN2, there is great translational possibility for the disease. Very few other diseases have such an obvious route for drug therapy. Bringing together the leading SMA research experts from around the world to share unpublished data and promote open communication and collaboration greatly quickens the pace of SMA research that will lead toward a treatment for this devastating disease.
