Browsing 2013 MU theses - Freely available online by Identifier "https://hdl.handle.net/10355/44711"
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Defining the therapeutic window in spinal muscular atrophy :
(University of Missouri--Columbia, 2013)Spinal muscular atrophy (SMA) is caused by the loss of a single gene, survival motor neuron-1 (SMN1), which results in the rapid deterioration of motor neuron integrity and function, most often leading to infantile death. ...