Spinal Muscular Atrophy (SMA) is an inherited neuromuscular disease that leads to motor dysfunction and death. It affects one in 6,000 to 10,000 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 sensitive to lowered SMN protein levels, and this cellular defect leads to the loss of muscle function in the limbs, neck, and chest in thes patients. Currently there is no treatment for SMA. Researchers have suggested that it is one of the neurological diseases closest to treatment, due to the unique presence of a back-up gene called SMN2 that provides great therapeutic possibility. Cure SMA has sponsored the SMA Researcher Meeting for 19 years. In 2015, the conference will be held June 18 to 20 in Kansas City, MO at The Westin and Sheraton Crown Center. It is now the largest SMA research conference in the world with about 250 attendees each year. Basic researchers, clinicians, and industrial researchers all attend the conference, allowing for cross-disciplinary dialogue crucial to therapy development. The meeting is held simultaneously with the Annual SMA Conference, with 1,200 affected by SMA attending, providing researchers a unique opportunity to interact with the patients they are dedicated to helping. The overarching goal of the meeting is to provide a venue for SMA experts from around the world to share unpublished data and develop scientific collaborations to hasten the identification of a treatment for SMA. Conference presentations are organized into 3 major areas: clinical research, basic research, and translational research. This allows researchers from different scientific disciplines to communicate about SMA. In addition, a major focus of the 2015 meeting will be a special session on Emerging Trends in Motor Neuron Pathobiology. In recent years, there has been significant progress in identifying potential molecular mechanisms underlying motor neuron disease, and the intent of this session is to explore whether commonality exists across mechanisms involved in various motor neuron diseases and discuss specific relevance to SMA, in particular in identifying novel drug approaches. Importantly, a major goal at Cure SMA is to develop therapies to treat all types of SMA and at every stage of disease progression. Therefore, it is critical for the scientific community to consider whether novel molecular approaches, beyond SMN regulation, should be pursued for the disease. An important secondary goal is to introduce new scientists to SMA research, including researchers in training to help build the future of our research community and industrial researchers new to SMA to help integrate them quickly and efficiently into our research community.
Spinal Muscular Atrophy (SMA) is a neuromuscular disease affecting one in 6,000 to 10,000 live births. It is the leading genetic killer of children under tw years of age. SMA is caused by mutations in the SMN1 gene, which is deleted in over 95% of patients. Currently there is no treatment for SMA, resulting in a large unmet medical need throughout the world. However, researchers have suggested that SMA is one of the neurological diseases closest to treatment. This is due both to the genetic homogeneity of the disease and the presence of a back-up gene called SMN2. This provides great therapeutic possibility for SMA, with few other diseases having such an obvious route for drug therapy. Cure SMA has hosted the Annual SMA Researcher Meeting for 19 years, with the goal of accelerating research towards a treatment for SMA. The conference is now the largest SMA research conference in the world with about 250 attendees each year. The overarching meeting goal is to provide a venue for SMA experts from around the world to share unpublished data and develop scientific collaborations to hasten the identification of a treatment for SMA. Conference presentations are organized into 3 major areas: clinical research, basic research, and translational research, because communication among researchers from these different scientific disciplines is critical to therapy development. A major focus of the 2015 meeting will b a special session on 'Emerging Trends in Motor Neuron Pathobiology'. In recent years, there has been significant progress in identifying potential molecular mechanisms underlying motor neuron disease, and the intent of this session is to explore whether commonality exists across mechanisms involved in various motor neuron diseases and discuss specific relevance to SMA, in particular in identifying novel drug approaches. A major goal for Cure SMA is to ensure therapies are developed to treat all types of SMA and at every stage of disease progression. Therefore, it is critical for our community to consider whether novel molecular approaches, beyond SMN regulation, should also be pursued for treating SMA. Finally, eight additional conference sessions on the newest findings in the areas of SMA basic research, clinical research, and drug development will also be held.
