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Cat No. | Product Name | Synonyms | Targets |
---|---|---|---|
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 |
Branaplam (LMI 070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator. | |||
T73475 | SMN-C2 | DNA/RNA Synthesis | |
SMN-C2 is a selective regulator of SMN2 gene splicing, a risdiplam analogue, a selective RNA-binding ligand that regulates pre-mRNA splicing and acts by binding to SMN2 pre-mRNA.SMN-C2 has the potential to be used in the... | |||
T12718L | RG7800 | RO6885247 | Others , DNA/RNA Synthesis |
RG7800 (RO6885247) has the potential for spinal muscular atrophy treatment. RG7800 is an SMN2 splicing modifier. | |||
T16757 | Risdiplam | RO7034067,RG7916 | DNA/RNA Synthesis |
Risdiplam (RO7034067) (RG7916) is orally administered. Risdiplam is a centrally and peripherally distributed SMN2 pre-mRNA splicing modifier which increases survival motor neuron (SMN) protein levels[1]. | |||
T12935 | SMN-C3 | MV8T2MCK57 | DNA/RNA Synthesis |
SMN-C3 (MV8T2MCK57) is an orally active modulator of SMN2 splicing, and has the potential to treat spinal muscular atrophy (SMA). | |||
T12718 | RG7800 hydrochloride (1449598-06-4 free base) | RO6885247 hydrochloride,RG7800 hydrochloride | Others |
RG7800 hydrochloride is an orally active modulator of SMN2 splicing. | |||
T64288 | RG7800 hydrochloride | ||
RG7800 hydrochloride is an orally active SMN2 splicing modulator that acts on SMN2 splicing (EC1.5x: 23 nM) and SMN proteins (EC1.5x: 87 nM). | |||
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. | |||
T78046 | RG7800 tetrahydrochloride | RO6885247 tetrahydrochloride | DNA/RNA Synthesis |
RG7800 hydrochloride is an orally active modulator of SMN2 splicing, exhibiting EC50 values of 23 nM for SMN2 splicing and 87 nM for SMN protein production. This compound holds promise as a therapeutic agent for spinal m... | |||
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 myoblas... | |||
T13723 | Homocarbonyltopsentin | PK4C9 | 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, an antisense oligonucleotide drug, modifies the pre-messenger RNA (pre-mRNA) splicing of the SMN2 gene, thereby enhancing the production of the full-length Survival Motor Neuron (SMN) protein. | |||
T62381 | Branaplam hydrochloride | ||
Branaplam (LMI070; NVS-SM1) hydrochloride is a selective, orally active SMN2 splicing modulator that acts on SMN (EC50: 20 nM). branaplam hydrochloride inhibits hERG (IC50: 6.3 μM). In a mouse model of severe spinal musc... |