[CAS NO. 1173755-55-9] Eteplirsen
PRODUCTS SPECIFICATIONS [1173755-55-9]
DESCRIPTION [1173755-55-9]
Overview
| MDL | - |
|---|---|
| Molecular Weight | 10306.00 |
| Molecular Formula | - |
| SMILES | [Eteplirsen] |
Summary
Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research [1] .
In Vitro
Eteplirsen acts to promote dystrophin production by restoring the translational reading frame of Duchenne muscular dystrophy (DMD) through specific skipping of exon 51 in defective gene variants [1] .
MCE has not independently confirmed the accuracy of these methods. They are for reference only.
Clinical Trial
| NCT Number | Sponsor | Condition | Start Date | Phase |
|---|---|---|---|---|
| NCT03992430 | Sarepta Therapeutics, Inc. |
Muscular Dystrophy, Duchenne
|
July 13, 2020 | Phase 3 |
| NCT01396239 | Sarepta Therapeutics, Inc. |
Duchenne Muscular Dystrophy
|
July 2011 | Phase 2 |
| NCT02420379 | Sarepta Therapeutics, Inc. |
Duchenne Muscular Dystrophy (DMD)
|
June 30, 2015 | Phase 2 |
| NCT02286947 | Sarepta Therapeutics, Inc. |
Muscular Dystrophy, Duchenne
|
November 2014 | Phase 2 |
| NCT00159250 | Imperial College London|Department of Health, United Kingdom|Sarepta Therapeutics, Inc. |
Duchenne Muscular Dystrophy
|
October 26, 2007 | Phase 1|Phase 2 |
| NCT00844597 | Sarepta Therapeutics, Inc.|British Medical Research Council |
Duchenne Muscular Dystrophy
|
January 2009 | Phase 1|Phase 2 |
| NCT03218995 | Sarepta Therapeutics, Inc. |
Duchenne Muscular Dystrophy
|
August 16, 2017 | Phase 2 |
| NCT03985878 | Sarepta Therapeutics, Inc. |
Duchenne Muscular Dystrophy
|
June 26, 2019 | Phase 2 |
| NCT02255552 | Sarepta Therapeutics, Inc. |
Duchenne Muscular Dystrophy (DMD)
|
November 17, 2014 | Phase 3 |
| NCT04179409 | Kevin Flanigan|Sarepta Therapeutics, Inc.|Nationwide Children´s Hospital |
Duchenne Muscular Dystrophy
|
February 18, 2020 | Phase 2 |
| NCT01540409 | Sarepta Therapeutics, Inc. |
Duchenne Muscular Dystrophy (DMD)
|
February 27, 2012 | Phase 2 |
Appearance
Solid
Shipping
Room temperature in continental US; may vary elsewhere.
Storage
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
Solvent & Solubility
DMSO : 50 mg/mL ( 4.85 mM ; Need ultrasonic)
Stock Solutions
| Concentration Solvent Mass | 1 mg | 5 mg | 10 mg |
|---|
| 1 mM | 0.0970 mL | 0.4852 mL | 0.9703 mL |
| 5 mM | --- | --- | --- |
| 10 mM | --- | --- | --- |
-
1.
Add each solvent one by one: PBS
Solubility: 33.33 mg/mL (3.23 mM); Clear solution; Need ultrasonic