Branaplam hydrochloride

SKU: orb1908994

Description

Branaplam hydrochloride (LMI070; NVS-SM1) is a selective and orally active SMN2 splicing modulator that acts directly on SMN with an EC50 value of 20 nM. Branaplam hydrochloride also inhibits hERG channels, demonstrating an IC50 of 6.3 μM. In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride significantly increased full-length SMN protein levels and markedly improved survival.

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Key Properties

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CAS Number	1562338-39-9
MW	429.95
Purity	98.45%
Formula	C₂₂H₂₈ClN₅O₂
SMILES	Cl.OC1=CC(=CC=C1C=2N=NC(OC3CC(NC(C)(C)C3)(C)C)=CC2)C=4C=NNC4
Target	Potassium Channel,Others,DNA/RNA Synthesis

Storage & Handling

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Storage	-20°C
Disclaimer	For research use only

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Potassium Channel,Others,DNA/RNA Synthesis

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