Drisapersen

Drisapersen, 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

[1251830-50-8]

Product Name Alternative

Kyndrisa; GSK2402968A; PRO051

UNSPSC

12352208

Target

DNA/RNA Synthesis; Dystrophin

Type

Oligonucleotides

Related Pathways

Cell Cycle/DNA Damage; Cytoskeleton

Field of Research

Others

Assay Protocol

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

Purity

97.73

Solubility

H2O : 100 mg/mL (ultrasonic)

Smiles

[Drisapersen]

Molecular Formula

C211H275N76O119P19S19

Molecular Weight

6978.00

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.