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Nusinersen
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Catalog No. T38656Cas No. 1258984-36-9
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.
Nusinersen
🥰Excellent
Catalog No. T38656Cas No. 1258984-36-9
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.
| Pack Size | Price | Availability | Quantity |
|---|---|---|---|
| 1 mg | $347 | In Stock | |
| 5 mg | $987 | In Stock | |
| 10 mg | $1,560 | In Stock | |
| 25 mg | $2,320 | In Stock | |
| 50 mg | $3,130 | In Stock |
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Product Introduction
Bioactivity
Chemical Properties
| Description | 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. |
| In vivo | In the ENDEAR phase 3 clinical trial, intrathecal administration of Nusinersen in infants with spinal muscular atrophy significantly improved outcomes. A motor milestone response was observed in 51% of treated infants (vs. 0% in controls), with increased event-free survival (HR = 0.53) and overall survival (HR = 0.37)[1].In severe SMA mouse models, a single subcutaneous injection of Nusinersen (30 μg) on postnatal day 1 significantly extended survival, improved motor neuron function, neuromuscular junction integrity, and motor behavior[2]. |
| Cas No. | 1258984-36-9 |
Storage & Solubility Information
| Storage | keep away from moisture | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
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(mother liquor concentration of 40 mg/mL), if you need to configure a concentration that exceeds the solubility of the product, please contact us first.Preparation method for in vivo formula: Take 50 μL DMSO main solution, add 300 μLPEG300 mix well and clarify, then add 50 more μL Tween 80, mix well and clarify, then add 600 more μLSaline/PBS/ddH2O mix well and clarify
main solution, add 300 μLPEG300 mix well and clarify, then add 50 more μL Tween 80, mix well and clarify, then add 600 more μLSaline/PBS/ddH2O mix well and clarifyFor Reference Only. Please develop an appropriate dissolution method based on your laboratory animals and route of administration.
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