Health
Effective Treatments for Spinal Muscle Atrophy
Spinal muscle atrophy or SMA is a genetic neuromuscular disorder. In this condition, the nerve cells in the brain and the spinal cord break down. Often, SMA affects babies and children, making it difficult for them to move their muscles properly. Although this progressive muscle weakness is rare, it is fatal universally. But, there is no absolute cure for SMA. The treatment options for spinal muscle atrophy mainly consist of various supportive measures like gene replacement therapy, medications, and other helpful methods. You can learn about those treatment approaches below. Nusinersen is a US Food and Drug Administration-approved medication for the treatment of SMA. It is a form of gene replacement therapy. The usage of this medication adjusts the amount of SMN2 genes. By improving the SMN2 gene’s splicing, this medication increases the amount of functional protein required for healthy motor neurons. The FDA has approved the use of this medicine for SMN types 1 to 4. The doctor may inject this medication several times. Onasemnogene abeparvovec-xioi is another form of gene therapy approved by the FDA as a treatment option for children less than two years of age with SMA. This gene therapy replaces the defective or missing survival motor neuron (SMN) 1 gene that creates the atrophy with a functional copy.
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