Spinal muscular atrophy type 1 is a genetic disorder that typically appears within the first six months of life, causing ...
who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively.
or patients with 5q SMA with a bi-allelic mutation in SMN1 and up to three copies of the SMN2 gene. Zolgensma is the world’s most expensive drug, with a price tag of more than $2.1 million per ...
The three diagnostic tests for identifying genetic mutations associated with SMA met new European Union regulatory ...
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