| Name | Branaplam |
| Description | Branaplam (LMI 070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator. |
| In vivo | To evaluate the efficacy of NVS-SM1, we used the SMNΔ7 mouse model, which displays a severe phenotype. In the specific colony used for this study, death typically occurs before postnatal day 15. We were pleased to observe that, in addition to a dose-dependent elevation of SMN protein in the brain, oral administration of NVS-SM1 improved body weight and extended lifespan, with 50% of the animals in the 1 mg per kg body weight group and 62% of animals in the 3 mg per kg body weight group showing increased survival. |
| Storage | Shipping with blue ice/Shipping at ambient temperature. |
| Solubility Information | DMSO : 3.96 mg/mL (10.06 mM), Sonication and heating are recommended.
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| Keywords | survival | splicing | spinal | SMN2 | SMN | SMA | RNASynthesis | RNA Synthesis | PotassiumChannel | Potassium Channel | orally | neuron-2 | muscular | motor | LMI-070 | LMI070 | KcsA | Inhibitor | inhibit | hERG | gene | DNASynthesis | DNA/RNA Synthesis | DNA Synthesis | Branaplam | atrophy |
| Inhibitors Related | 5-Fluorouracil | Adenine hemisulfate | Erythromycin thiocyanate | Guanidine hydrochloride | Hexane-1,6-diol | 1,8-Cineole | 1,4-Naphthoquinone | Adenine | Vidarabine | Carbazole | Thymidine | Usnic Acid |
| Related Compound Libraries | Highly Selective Inhibitor Library | Pain-Related Compound Library | Anti-Neurodegenerative Disease Compound Library | Bioactive Compound Library | Membrane Protein-targeted Compound Library | Anti-Cancer Clinical Compound Library | Drug Repurposing Compound Library | Anti-Aging Compound Library | Bioactive Compounds Library Max | Potassium Channel Targeted Library | Ion Channel Targeted Library | Anti-Cancer Drug Library |