Drisapersen (sodium)

Drisapersen sodium, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.

Product Specifications

CAS Number

[1181666-20-5]

Product Name Alternative

Kyndrisa (sodium) ; GSK2402968A (sodium) ; PRO051 (sodium)

UNSPSC

12352208

Target

Others

Type

Oligonucleotides

Related Pathways

Others

Field of Research

Others

Assay Protocol

https://www.medchemexpress.com/drisapersen-sodium.html

Purity

98.61

Solubility

10 mM in H2O

Smiles

[Drisapersen(sodium)]

Molecular Formula

C211H256N76Na19O119P19S19

Molecular Weight

6978 (free acid)

References & Citations

[1]Flanigan KM, Voit T, Rosales XQ, et al. Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial. Neuromuscul Disord. 2014;24 (1) :16-24. |[2]Voit T, Topaloglu H, Straub V, et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II) : an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol. 2014;13 (10) :987-996.