Nusinersen (nusinersen) is an antisense oligonucleotide (ASO) for the treatment of pediatric and adult spinal muscular atrophy (SMA) that alters the way the SMN2 gene is processed and increases the production of SMN proteins, which are deficient in SMA patients.
Reinheit:
98.62%
CAS Nummer:
[1258984-36-9]
Target-Kategorie:
DNA/RNA Synthesis
T38656
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