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Cat. No.	Product Name	Target	Signaling Pathways
T34664	SMN2-Stablizer-27 SMN2-Stablizer 27,SMN2 Stablizer-27
	SMN2-Stablizer-27 is a stablizer of survival of motor neuron 2 (SMN2), and a post-translationally stablizing SMN protein for the treatment of spinal muscular atrophy (SMA).
T4641	Branaplam LMI 070,NVS-SM1	Others; Potassium Channel; DNA/RNA Synthesis	Cell Cycle/Checkpoint; DNA Damage/DNA Repair; Membrane transporter/Ion channel; Others
	Branaplam (LMI 070) 是一种高效、选择性和口服活性的小分子 SMN2 剪接调节剂，对 SMN 的 EC50为 20 nM。它可提高全长 SMN 蛋白并延长其生存期，也抑制 hERG，IC50为 6.3 μM。
T16757	Risdiplam RO7034067,RG7916	DNA/RNA Synthesis	Cell Cycle/Checkpoint; DNA Damage/DNA Repair
	Risdiplam (RO7034067) 是一种中枢和外周分布的 SMN2 前 mRNA 剪接修饰剂，可增加运动神经元存活 (SMN) 蛋白水平。
T12718L	RG7800 RO6885247	Others; DNA/RNA Synthesis	Cell Cycle/Checkpoint; DNA Damage/DNA Repair; Others
	RG7800 (RO6885247) 是一种 SMN2 拼接修饰剂，有用于治疗脊髓性肌萎缩的研究潜力。
T73475	SMN-C2	DNA/RNA Synthesis	Cell Cycle/Checkpoint; DNA Damage/DNA Repair
	SMN-C2 是一种选择性的 SMN2 基因剪接调节剂，是risdiplam类似物，是调节前 mRNA 剪接的选择性 RNA 结合配体，通过结合 SMN2 pre-mRNA 来发挥作用。SMN-C2 具有研究脊髓性肌萎缩症 (SMA) 的潜力。
T12935	SMN-C3 MV8T2MCK57	DNA/RNA Synthesis	Cell Cycle/Checkpoint; DNA Damage/DNA Repair
	SMN-C3 (MV8T2MCK57) 是口服有效的 SMN2 剪接调节剂。SMN-C3可用于脊椎肌肉萎缩 (SMA) 的研究。
T78046	RG7800 tetrahydrochloride RO6885247 tetrahydrochloride	DNA/RNA Synthesis	Cell Cycle/Checkpoint; DNA Damage/DNA Repair
	RG7800 hydrochloride为一可口服SMN2剪接调节剂，其对SMN2剪接及SMN蛋白的EC1.5x值分别为23 nM及87 nM。
T12718	RG7800 hydrochloride (1449598-06-4 free base) RO6885247 hydrochloride,RG7800 hydrochloride	Others	Others
	RG7800 hydrochloride is an orally active modulator of SMN2 splicing.
T26893	Branaplam mesylate (1562338-42-4 free base) LMI070,Branaplam mesylate,LMI 070,LMI-070
	Branaplam is a highly potent, selective and orally active SMN2 splicing modulator. Branaplam increases the amount of functional SMN protein produced by the SMN2 gene through modifying its splicing.
T64288	RG7800 hydrochloride
	RG7800 hydrochloride 是一种口服具有活力的 SMN2 剪接调节剂，能够作用于 SMN2 剪接 (EC1.5x: 23 nM) 及 SMN 蛋白 (EC1.5x: 87 nM)。
T13723	Homocarbonyltopsentin PK4C9	Others	Others
	Homocarbonyltopsentin is a small-molecule TSL2-binding compound, binds to pentaloop conformations of TSL2 and promotes a shift to triloop conformations that display enhanced SMN2 exon 7 (E7) splicing (EC50: 16 μM).
T38656	Nusinersen
	Nusinersen is an antisense oligonucleotide drug that modifies pre–messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein.
T33764	NVS-SM2
	NVS-SM2 is an effective small molecule splicing enhancer of SMN2 with oral activity. NVS-SM2 showed increased exon 7 inclusion and upregulated SMN protein expression in SMA fibroblasts and SMN cells Δ7 5025 mouse myoblasts.
T62381	Branaplam hydrochloride
	Branaplam (LMI070; NVS-SM1) hydrochloride 是一种选择性的、口服具有活力的 SMN2 拼接调节剂，能够作用于 SMN (EC50: 20 nM)。Branaplam hydrochloride 能够抑制 hERG (IC50: 6.3 μM)。在严重的脊髓性肌萎缩症 (SMA) 小鼠模型中，Branaplam hydrochloride 能够增加全长 SMN 蛋白并提高其生存期。

化合物

SMN2-Stablizer-27

Cat.No: T34664

Synonym: SMN2-Stablizer 27,SMN2 Stablizer-27

Cat.No: T4641

Synonym: LMI 070,NVS-SM1

Target: Others, Potassium Channel, DNA/RNA Synthesis

Cat.No: T16757

Synonym: RO7034067,RG7916

Target: DNA/RNA Synthesis

Cat.No: T12718L

Synonym: RO6885247

Target: Others, DNA/RNA Synthesis

Cat.No: T73475

Target: DNA/RNA Synthesis

Cat.No: T12935

Synonym: MV8T2MCK57

Target: DNA/RNA Synthesis

RG7800 tetrahydrochloride

Cat.No: T78046

Synonym: RO6885247 tetrahydrochloride

Target: DNA/RNA Synthesis

RG7800 hydrochloride (1449598-06-4 free base)

Cat.No: T12718

Synonym: RO6885247 hydrochloride,RG7800 hydrochloride

Target: Others

Branaplam mesylate (1562338-42-4 free base)

Cat.No: T26893

Synonym: LMI070,Branaplam mesylate,LMI 070,LMI-070

RG7800 hydrochloride

Cat.No: T64288

Homocarbonyltopsentin

Cat.No: T13723

Synonym: PK4C9

Target: Others

Cat.No: T38656

Cat.No: T33764

Branaplam hydrochloride

Cat.No: T62381

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