Branaplam (LMI070; NVS-SM1) hydrochloride is a highly potent, selective and orally activesurvival motor neuron-2 (SMN2)splicing modulator with anEC₅₀of 20 nM for SMN. Branaplam hydrochloride inhibitshuman-ether-a-go-go-related gene (hERG)with anIC₅₀of 6.3 μM. Branaplam hydrochloride elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model^[1][2].

Branaplam (LMI070; NVS-SM1) hydrochloride treatment induces changes in the levels of 175 genes in human fibroblasts^[1].

Branaplam (LMI070; NVS-SM1; 3, 10, 30 mg/kg; oral) hydrochloride produces dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord^[1].
Branaplam (1 mg/kg of IV; 3 mg/kg of PO) hydrochloride has a CL of 25 mL/min/kg and an AUC of 3.03 μMoh^[2].
A single Branaplam (oral; 30 mg/kg) hydrochloride results in significant and durable SMN protein elevation in brain for up to 160 hours in C/+ mice^[1].
Branaplam (oral; 0.03, 0.1, 0.3, 1, 3 mg/kg) hydrochloride improves body weight and extendes lifespan in n SMNΔ7 mice^[1].

429.94

Solid

C₂₂H₂₈ClN₅O₂

1562338-39-9

Room temperature in continental US; may vary elsewhere.

4°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

H₂O : 200 mg/mL(465.18 mM;Need ultrasonic)

请根据产品在不同溶剂中的溶解度选择合适的溶剂配制储备液；一旦配成溶液，请分装保存，避免反复冻融造成的产品失效。
储备液的保存方式和期限：-80℃, 6 months; -20℃, 1 month (sealed storage, away from moisture)。-80℃ 储存时，请在 6 个月内使用，-20℃ 储存时，请在 1 个月内使用。