Year 4/NCE

The goal of this project has now been reached. We have developed a new screening platform using motor neurons from induced pluripotent stem cells taken from children with spinal muscular atrophy. Through this technology we have screened thousands of compounds and have shown a small sub set that active gene expression and enhance motor neuron survival in this model. These compounds will now be moved to the next stage for validation. This funding has allowed us to complete the development of this tool/technology and put us in a strong position to continue these studies and the drug development process to move interesting drugs to the market for spinal muscular atrophy.