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.
Shipping Conditions
Blue Ice
Storage Conditions
-20°C (Powder, sealed storage, away from moisture)
Scientific Category
Oligonucleotides
Clinical Information
Phase 3
Available Sizes
Frequently Asked Questions
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