Spinal Muscular Atrophy (SMA) is a genetic disorder affecting motor neurons in the spinal cord, resulting in muscle weakness and paralysis. It is inherited and caused by a mutation in the SMN1 gene. SMA has five types based on age of onset and physical milestones reached. Treatments for SMA include medications like Nusinersen and Onasemnogene abeparvovec, as well as supportive therapies like physical therapy and respiratory care. Living with SMA can be complex, but with the right support, individuals can lead fulfilling lives. Advocates are working to improve access to treatment and supportive care for individuals with SMA.
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What is Spinal Muscular Atrophy?
