Another important milestone was achieved by the promising Spinal Muscular Atrophy (SMA) stem cell program today: California Stem Cell (CSC) received orphan drug designation from the FDA to its stem cell-derived motor neuron product aimed at treating SMA. This provides several incentives to companies in the private sector, such as CSC, developing novel drugs or biologics to treat diseases with relatively small market potential. This is also extremely important as reaching this milestone is a positive step forward for the SMA community and shows significant progress being made to develop an effective treatment for our children.
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