Columbia receives up to $15 million from Spinal Muscular Atrophy Foundation

This is a remarkable time in SMA research, said Gerald D. Fischbach, M.D., executive vice president for Health and Biomedical Sciences and dean of the Faculty of Medicine, Columbia University Medical Center. A decade ago we knew very little about what causes SMA, and now we know the gene that causes the disease. The SMA Foundation funding will allow our Center for Motor Neuron Biology and Disease to rapidly advance the knowledge of how this gene causes motor neurons to degenerate, and turn this into improved clinical care for patients.

The gift, which will be distributed over five years, is the largest ever made by a private foundation for SMA research. The grant symbolizes a new type of funding between private foundations and universities active funding, a style popularized by the Gates Foundation. As part of the grant agreement, the Center for Motor Neuron Biology and Disease will recruit new investigators to complement existing Columbia expertise and is incentivized to reach specific scientific goals.

The SMA Foundation is a tremendous partner in our work, said Christopher E. Henderson, Ph.D., co-director of the Center for Motor Neuron Biology and Disease, along with Serge Przedborski, M.D., Ph.D., professor of neurology, pathology and cell biology, and Darryl De Vivo, M.D., the Sidney Carter Professor of Neurology. We value their ongoing input and collaboration, which provide a new perspective on how to take basic research findings and move them toward the clinic. We hope that this approach will generate both new biological insights and relief for families living with SMA.

"We have known about SMA for more than a century; however, there remain significant gaps in our knowledge about this disease. We need to fill in these gaps in order to develop effective treatments," said Dr. De Vivo, who is also director of the SMA Clinic at Columbia University Medical Center. Although it is the most common genetic cause of infant death and is

Contact: Elizabeth Streich
Columbia University Medical Center

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