Spinal Muscular Dystrophy Pipeline
May 12, 2025
Spinal Muscular Dystrophy Treatment: A Deep Dive into Current Therapies and Future Prospects
Spinal muscular atrophy (SMA) is the second most prevalent autosomal recessive disorder, with a carrier frequency of approximately 1 in 40 to 50 individuals and an estimated occurrence of 1 in 10,000 live births. The majority of SMA cases are caused by the deletion of a portion of the SMN1 gene, which impairs the p...
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