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Medchemexpress LLC Branaplam hydrochloride | 1562338-39-9 | 99.4% | 429.94 | 1 ML
SDP

Supplier:  Medchemexpress LLC HY19620A10MM/1ML

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Branaplam (LMI070; NVS-SM1) hydrochloride is a highly potent, selective, and orally active survival motor neuron-2 (SMN2) splicing modulator. It has an EC50 of 20 nM for SMN and inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. This compound is capable of elevating full-length SMN protein and extending survival in a severe spinal muscular atrophy (SMA) mouse model. It is intended for research use only and not for sale to patients.

  • Highly potent, selective, and orally active SMN2 splicing modulator.
  • Elevates full-length SMN protein.
  • Extends survival in a severe spinal muscular atrophy (SMA) mouse model.
  • Produces dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord.
  • Improves body weight and extends lifespan in n SMN∆7 mice.

Catalog No. 50-004-01271


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