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Cat. No.	Product Name	Target	Signaling Pathways
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。
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。
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。
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).
T16902	SMND-309	Others	Others
	SMND-309 is a metabolite of salvianolic acid B. It also shows neuroprotective effects in cultured neurons and in permanent middle cerebral artery occlusion rats.
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。
T3336	Rg3039 PF-06687859	Others	Others
	Rg3039 (PF-06687859) 是一种具有口服活性、能够透过血脑屏障的DcpS 抑制剂，其IC50=0.069 nM。
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.
T35594	Cuspin-1
	The Survival of Motor Neurons (SMN) protein participates in RNA splicing. Decreases in SMN, typically a consequence of defects in the smn1 gene, result in the death of motor neurons and lead to the neurodegenerative disease, spinal muscular atrophy (SMA). Cuspin-1 is a small molecule upregulator of SMN that has been shown in vitro to increase levels of SMN in SMA patient fibroblasts by 50% at 18 μM. Its mechanism of action is thought to involve increased phosphorylation of ERK to initiate Ras-Ra...
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.
T28067	ML372 ML 372,ML-372
	ML372 is a potent and selective SMN Modulator (EC50 = 12 nM, 325% increase inSMN2) with good potency, pharmacokinetics, tolerance, and CNS penetration.
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。
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。
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)。
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.
T12718	RG7800 hydrochloride (1449598-06-4 free base) RO6885247 hydrochloride,RG7800 hydrochloride	Others	Others
	RG7800 hydrochloride is an orally active modulator of SMN2 splicing.

化合物

Cat.No: T12935

Synonym: MV8T2MCK57

Target: DNA/RNA Synthesis

Cat.No: T73475

Target: DNA/RNA Synthesis

Cat.No: T4641

Synonym: LMI 070,NVS-SM1

Target: Others, Potassium Channel, DNA/RNA Synthesis

SMN2-Stablizer-27

Cat.No: T34664

Synonym: SMN2-Stablizer 27,SMN2 Stablizer-27

Cat.No: T16902

Target: Others

Cat.No: T16757

Synonym: RO7034067,RG7916

Target: DNA/RNA Synthesis

Cat.No: T3336

Synonym: PF-06687859

Target: Others

Cat.No: T33764

Cat.No: T35594

Branaplam mesylate (1562338-42-4 free base)

Cat.No: T26893

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

Cat.No: T28067

Synonym: ML 372,ML-372

RG7800 tetrahydrochloride

Cat.No: T78046

Synonym: RO6885247 tetrahydrochloride

Target: DNA/RNA Synthesis

Branaplam hydrochloride

Cat.No: T62381

RG7800 hydrochloride

Cat.No: T64288

Cat.No: T38656

RG7800 hydrochloride (1449598-06-4 free base)

Cat.No: T12718

Synonym: RO6885247 hydrochloride,RG7800 hydrochloride

Target: Others

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