What is currently being done?
NIH has selected SMA as a model for a new approach to funding translational research. Translational research develops findings made by scientists in the lab into drugs and treatments that doctors can use to save the lives of patients. SMA was chosen due to the fact that it 1) offers a high probability of developing treatment or cure, 2) is relatively common, 3) is a devastating children’s disease, and 4) has no current treatment. As a result of its investment in the translational research project, NINDS funding for SMA research has reached approximately $13 million annually. [For more information about the NINDS translational research project, please visit the SMA Project website.] Additional funding for investigators is provided by patient advocacy organizations in the US and worldwide. These funds support investigators at world-renowned research centers, including University of Pennsylvania, Johns Hopkins, Columbia University, Ohio State, MIT, University of California, Arizona State,
NIH has selected SMA as a model for a new approach to funding translational research. Translational research develops findings made by scientists in the lab into drugs and treatments that doctors can use to save the lives of patients. SMA was chosen due to the fact that it 1) offers a high probability of developing treatment or cure, 2) is relatively common, 3) is a devastating children’s disease, and 4) has no current treatment. The program is proceeding well and the next step will be to advance compounds from the SMA translation pilot into the clinic trails.
