What Happens When Spinraza Activates Motor Neurons?
When people ask what happens when Spinraza “activates” motor neurons, the more accurate explanation is that Spinraza does not switch dead motor neurons back on. Instead, Spinraza, also known as nusinersen, helps the backup SMN2 gene produce more full-length SMN protein, giving surviving motor neurons more of the protein they need to function. Spinraza increases the inclusion of exon 7 in SMN2 messenger RNA, resulting in greater production of full-length SMN protein.
As SMN protein production increases, surviving motor neurons may be better supported in their ability to send signals from the central nervous system to the muscles. Those signals are essential for movement, posture, breathing, swallowing, and other physical functions affected by Spinal Muscular Atrophy. Motor neurons require SMN protein to work and that when motor neurons are lost, muscles receive fewer signals and become progressively weaker. This is why Spinraza is considered a disease-modifying treatment: it targets an underlying biological problem in 5q SMA rather than simply treating individual symptoms. However, treatment cannot necessarily restore motor neurons that have already been permanently lost, which is one reason early diagnosis and timely treatment are so important.
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