This product is for research use only, not for human use. We do not sell to patients.

Size | Price | Stock |
---|---|---|
25mg | $410 | 3-6 Days |
50mg | $680 | 3-6 Days |
100mg | $950 | 3-6 Days |
250mg | $1390 | 3-6 Days |
500mg | $1960 | 3-6 Days |
Cat #: V3853 CAS #: 1562338-39-9 Purity ≥ 99%
Description: Branaplam HCl (LMI-070 HCl; NVS-SM1) is a potent, orally bioactive, small-molecule enhancer of SMN2 (survival of motor neuron-2) splicing that elevates full-length SMN protein and extends survival in a severe SMA mouse model. The transient double-strand RNA structure created by the U1 small nuclear ribonucleic protein (snRNP) complex and SMN2 pre-mRNA is stabilized as the molecular mechanism of action. Separate from constitutive recognition, the binding affinity of U1 snRNP to the 5' splice site is enhanced in a sequence-specific manner. This novel mechanism highlights the viability of sequence-selective splice modulation mediated by small molecules and the possibility of applying this tactic to other splicing disorders.
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- Physicochemical and Storage Information
- Protocol
- Related Biological Data
- Stock Solution Preparation
- Quality Control Documentation
Molecular Weight (MW) | 429.94 |
---|---|
Molecular Formula | C22H28ClN5O2 |
CAS No. | 1562338-39-9 |
Storage | Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment (e.g. under nitrogen), avoid exposure to moisture and light. |
Synonyms | LMI070 HCl; NVS SM1 hydrochloride; LMI 070; NVS-SM1 HCl; LMI-070; NVSSM1 hydrochloride |
Solvent volume to be added | Mass (the weight of a compound) | |||
---|---|---|---|---|
Mother liquor concentration | 1mg | 5mg | 10mg | 20mg |
1mM | 2.3259 mL | 11.6295 mL | 23.2591 mL | 46.5181 mL |
5mM | 0.4652 mL | 2.3259 mL | 4.6518 mL | 9.3036 mL |
10mM | 0.2326 mL | 1.1630 mL | 2.3259 mL | 4.6518 mL |
20mM | 0.1163 mL | 0.5815 mL | 1.1630 mL | 2.3259 mL |
This equation is commonly abbreviated as: C1 V1 = C2 V2
- (1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
- (2) Be sure to add the solvent(s) in order.