The three diagnostic tests for identifying genetic mutations associated with SMA met new European Union regulatory ...
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Real-world data fill knowledge gap to assess treatment options for infants with spinal muscular atrophyAlthough almost all cases of SMA have the same underlying genetic cause—a biallelic deletion or mutation in the SMN1 gene—clinical ... of Philadelphia Infant Test of Neuromuscular Disorders ...
The AveXis approach to SMA is to replace the SMN1 gene in the motor neurons of the central nervous system. We have seen success in this endeavor both scientifically and clinically. Building on ...
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