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Abstract
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease characterized by high incidence and severity. While the disease affects approximately 1 in 6,000-10,000 live births, the carrier frequency is observed at a rate of 1 in 40 to 1 in 60 individuals. SMA is the leading genetic cause of infant mortality. This review explores advancements in gene and RNA therapies for both advanced and developing stages of SMA, considering the evolving landscape of biotechnology.
