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Branaplam

Cat. No. M13649

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Branaplam Structure
Synonym:

LMI070; NVS-SM1

Size Price Availability Quantity
5mg USD 120  USD120 In stock
10mg USD 200  USD200 In stock
25mg USD 420  USD420 In stock
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Biological Activity

Branaplam (LMI070; NVS-SM1) is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model.

Chemical Information
Molecular Weight 393.48
Formula C22H27N5O2
CAS Number 1562338-42-4
Solubility (25°C) DMSO 4 mg/mL
Storage 2-8°C, dry, sealed
References

[1] Diethilde Theil, et al. Biol Open. Orally administered branaplam does not impact neurogenesis in juvenile mice, rats, and dogs

[2] Ravindra N Singh, et al. Neurosci Insights. The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy

[3] Ai-Ming Yu, et al. Pharmacol Rev. RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges

[4] Janbernd Kirschner, et al. Eur J Paediatr Neurol. European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy

[5] Sithara Ramdas, et al. Expert Opin Pharmacother. New treatments in spinal muscular atrophy: an overview of currently available data

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Keywords: Branaplam, LMI070; NVS-SM1 supplier, DNA/RNA Synthesis, inhibitors, activators

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