SMA is a neurodegenerative disorder that—left untreated—can result in progressive muscular atrophy, and in its most severe forms, premature death1,2 Spinal muscular atrophy (SMA) is a rare genetic disease caused by the deletion or mutation of the survival motor neuron 1 (SMN1) gene. The SMN1 gene produces survival motor neuron (SMN) protein that is critical … Continue reading What is SMA? […]
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