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This product is for research use only and is not for human consumption or therapeutic use.
CAS Number:
1562338-42-4
Purity:
≥98%
Formulation:
A crystalline solid
Molecular Weight:
393.5
Alternative Names:
5-(1h-pyrazol-4-yl)-2-[6-[(2,2,6,6-tetramethyl-4-piperidinyl)oxy]-3-pyridazinyl]-phenol, branaplam, nvs-sm1
- Data sheet: View or download
- MSDS: View or download
Product Description:
An SMN2 splice modulator; enhances SMN2 splicing activity by stabilizing the snRNP-SMN2 pre-mRNA complex; increases the expression of the full-length SMN2 transcript at 3-20 mg/kg; increases SMN protein levels in the brain and spinal cord in the C/+ mouse model of SMA at 0.3-30 mg/kg; increases survival of SMNΔ7 mice at 1 and 3 mg/kg
Storage Temperature:
-20°C