Product Description: 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.
Formula: C211H275N76O119P19S19
References: [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.
CAS Number: 1251830-50-8
Molecular Weight: 6978.00
Compound Purity: 96.05
Research Area: Others
Solubility: H2O : 100 mg/mL (ultrasonic)
Target: DNA/RNA Synthesis;Dystrophin
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