Spinal Muscular Atropy (Sma) In Children And Current Therapies
Children diagnosed with SMA disease may be cured by the modification of the defective SMN1 gene. SMA, which affects children's functional status from early infancy through adulthood and is the second most prevalent fatal autosomal recessive condition in children, is the second most common cause of death in children. It is probable that a delayed diagnosis will prevent patients with SMA from receiving the most effective early treatment available, despite the fact that the nature of the functional loss that occurs during the wait is unknown.
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