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Life Saving Spinal Muscular Atrophy (SMA) Treatment | Sam's Story
Spinal muscular atrophy (SMA) is a hereditary disease that causes progressive weakness and can lead to death. Before a gene therapy medication was developed that changes the way the broken gene in SMA is expressed, there was no way to slow the progression of weakness. Thanks to a huge team effort at Johns Hopkins, this life saving medication ...
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