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Results for "

SMA

" in MedChemExpress (MCE) Product Catalog:

18

Inhibitors & Agonists

1

Screening Libraries

1

MCE Kits

1

Inhibitory Antibodies

1

Recombinant Proteins

3

Antibodies

Cat. No. Product Name Target Research Areas Chemical Structure
  • HY-124648

    DNA/RNA Synthesis Inflammation/Immunology
    SMN-C2, an analog of RG-7916, is a selective modulator of SMN2 gene splicing that acts by binding SMN2 pre-mRNA, thereby increasing far upstream element binding protein 1 (FUBP1) and KH-spliced RNA binding Protein affinity regulator protein (KHSRP) to the SMN2 pre-mRNA complex. SMN-C2 can be used in spinal muscular atrophy (SMA) research .
    SMN-C2
  • HY-124713

    DNA/RNA Synthesis Neurological Disease
    ML372 inhibits survival motor neuron (SMN) protein ubiquitination, increases SMN protein stability without affecting mRNA expression. ML372 improves spinal muscular atrophy (SMA) in mice. ML372 is brain penetrant and has a reasonable exposure and half-life in vivo .
    ML372
  • HY-19620
    Branaplam
    Maximum Cited Publications
    9 Publications Verification

    LMI070; NVS-SM1

    DNA/RNA Synthesis Potassium Channel Cancer
    Branaplam (LMI070; NVS-SM1) is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model .
    Branaplam
  • HY-19620A
    Branaplam hydrochloride
    Maximum Cited Publications
    9 Publications Verification

    LMI070 hydrochloride; NVS-SM1 hydrochloride

    DNA/RNA Synthesis Potassium Channel Cancer
    Branaplam (LMI070; NVS-SM1) hydrochloride is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam hydrochloride inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam hydrochloride elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model .
    Branaplam hydrochloride
  • HY-155106

    PKG Cardiovascular Disease
    SMA4 is a selective PKG1α activator with basal EC50 value of 29 μM. SMA4 facilitates phosphorylation of the known PKG1 substrate, vasodilator-stimulated phosphoprotein (VASP), and inhibits human pulmonary arterial smooth muscle cells (hPASMC) proliferation. SMA4 can be used for cardiovascular disease research .
    <em>SMA</em>4
  • HY-112633

    DNA/RNA Synthesis Others
    SMN-C3 is an orally active SMN2 splicing modulator and has the potential to treat spinal muscular atrophy (SMA).
    SMN-C3
  • HY-112980

    DNA/RNA Synthesis Inflammation/Immunology
    Nusinersen is an antisense oligonucleotide agent that modifies pre–messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein .
    Nusinersen
  • HY-112980A

    DNA/RNA Synthesis Inflammation/Immunology
    Nusinersen sodium is an antisense oligonucleotide agent that modifies pre–messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein .
    Nusinersen sodium
  • HY-133897
    (Rac)-Indoximod
    1 Publications Verification

    1-Methyl-DL-tryptophan; (Rac)-NLG-8189

    Indoleamine 2,3-Dioxygenase (IDO) Apoptosis Inflammation/Immunology Cancer
    (Rac)-Indoximod (1-Methyl-DL-tryptophan) is an indoleamine 2,3-dioxygenase (IDO) inhibitor. Co-treatment with IFN-γ and (Rac)-Indoximod markedly reduces the activity of human cardiac myofibroblasts (hCMs) expressing α-SMA and induces apoptosis through up-regulating the IRF-1, Fas, and FasL genes .
    (Rac)-Indoximod
  • HY-143237

    Phosphodiesterase (PDE) Others
    ATX inhibitor 11 (compound 13c) is a potent ATX (autotaxin) inhibitor, with an IC50 of 2.7 nM. ATX inhibitor 11 can typically alleviate the severity of fibrosis tissues and effectively reduce the deposition of fibrotic biomarker α-SMA in mice fibrosis model. ATX inhibitor 11 can be used for lung fibrosis research .
    ATX inhibitor 11
  • HY-151932

    FXR Inflammation/Immunology
    FXR agonist 3 is an anti-NASH agent, acting by activating FXR. FXR agonist 3 inhibits COL1A1, TGF-β1, α-SMA and TIMP1 expression with anti-fibrogenic activity. FXR agonist 3 significantly reduces liver steatosis and inflammation, improves liver fibrosis level .
    FXR agonist 3
  • HY-100446

    NTU281 is a potent transglutaminase-2 inhibitor. NTU281 can reduce the increases in serum creatinine and albuminuria in diabetic rats. NTU281 can also reduce glomerular collagen I accumulation, Hic-5 and α-SMA expression, and apoptosis. NTU281 can be used for researching glomerulosclerosis caused by diabetes .
    NTU281
  • HY-151427

    TGF-β Receptor Inflammation/Immunology
    TGFβ1-IN-1 (compound 42) is a potent, orally active TGF-β1 inhibitor. TGFβ1-IN-1 inhibits the upregulation of TGF-β1-induced fibrosis markers (α-SMA and fibronectin) and can be used in liver fibrosis disease studies .
    TGFβ1-IN-1
  • HY-124293
    AA147
    3 Publications Verification

    ATF6 Reactive Oxygen Species Neurological Disease Metabolic Disease
    AA147 is a endoplasmic reticulum (ER) proteostasis regulator. AA147 promotes protection against oxidative damage in neuronal cells and prevents endothelial barrier dysfunction by activating ATF6 arm (selectively) of the unfolded protein response (UPR) and the NRF2 oxidative stress response. AA147 can rebalances XBP1s expression in vivo, and also induces survival motor neuron (SMN) expression and spinal motorneuron (MN) protection .
    AA147
  • HY-111055

    GSK-3 Neurological Disease
    BIP-135 is a potent and selective ATP-competitive GSK-3 inhibitor, with IC50s of 16 nM and 21 nM for GSK-3α and GSK-3β, respectively. BIP 135 exhibits neuroprotective effect .
    BIP-135
  • HY-111520

    DNA/RNA Synthesis Neurological Disease
    NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NVS-SM2 can be used for spinal muscular atrophy (SMA) research .
    NVS-SM2
  • HY-162147

    mTOR Autophagy Inflammation/Immunology
    Nur77 modulator 3 (9e) can bind to Nur77 and inhibit TGF-β1-induced α-SMA and COLA1 expression in a Nur77-dependent manner. Nur77 modulator 3 induces Nur77 expression and enhances autophagic flux by inhibiting the mTORC1 signaling pathway in vitro and in vivo. Nur77 modulator 3 blocks the progression of hepatic fibrosis .
    Nur77 modulator 3
  • HY-146434

    TGF-beta/Smad Inflammation/Immunology
    TGFβ-IN-2 (Compound 9d) inhibits TGF-β-induced total collagen accumulation in NRK-49F cells with the IC50 of 4.31 μM. TGFβ-IN-2 suppresses the TGF-β-induced protein expression of COL1A1, α-SMA, and p-Smad3 in vitro. TGFβ-IN-2 can be used as a potential effective compound for anti-fibrosis in vivo by oral administration .
    TGFβ-IN-2

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