The Global Spinal Muscular Atrophy Market is estimated to driven by Increased screening and early diagnosis
Spinal muscular atrophy (SMA) is a genetic disorder that affects the motor nerve cells in the spinal cord, resulting in weakness and wasting of muscles. SMA is caused by a mutation or deficiency of the survival motor neuron 1 (SMN1) gene. There are three types of SMA - type I being the most severe form that begins within the first 6 months of life. Treatment options for SMA include Nusinersen, onasemnogene abeparvovec, and risdiplam. Nusinersen is the first approved treatment for SMA, and works by modifying splicing
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