In an exclusive interview, SMA News Today Senior Columnist Maureen Newman spoke with Dr. Adrian Krainer of Cold Spring Harbor Laboratory. In conjunction with Isis Pharmaceuticals, Dr. Krainer and his team are making promising strides into better understanding how SMA works. In January, Dr. Adrian Krainer of Cold Spring Harbor Laboratory…
To better understand spinal muscular atrophy (SMA), scientists must conduct studies using more than just cells in a culture dish. It is best to recapitulate the human body using a large animal model, but many scientists use mice due to their availability. To fill the gap of large animal…
Spinal muscular atrophy (SMA) is among the many motor neuron diseases that have no cure. One reason for the lack of cures is the inexistence of reliable in vitro disease models to be used as platforms for research and development of new drugs. This is partially attributed  to the difficulty in…
A new approach to treating spinal muscular atrophy (SMA) is in the works in the laboratory of Matthew E. R. Butchbach at the Nemours Alfred I. duPont Hospital for Children in Wilmington, Delaware. The team of researchers, which includes Drs. Sean G. Mack, Daniel J. Cook, and Prasad Dhurjati,…
Encouraging news from Isis Pharmaceuticals, Inc., presented at the 19th International World Muscle Society Congress in Berlin, demonstrated the efficacy of ISIS-SMNRx in treating spinal muscular atrophy (SMA) in infants and children. Results were from the ongoing open-label Phase 2 clinical trial. Isis is also enrolling patients in the…
Parents want what is best for their children, and parents of children with spinal muscular atrophy (SMA) are no exception. At times, these parents can feel stressed about their children’s medical condition, and any way that stress can be alleviated is vital to improving parent outlook on managing their children’s…
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