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Results for "

spinal muscular atrophy

" in MCE Product Catalog:

11

Inhibitors & Agonists

1

Screening Libraries

1

Isotope-Labeled Compounds

Cat. No. Product Name Target Research Areas
  • HY-124713
    ML372

    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.
  • HY-111520
    NVS-SM2

    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.
  • HY-19620A
    Branaplam hydrochloride

    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.
  • HY-19620
    Branaplam

    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.
  • HY-B1104
    Indoprofen

    (±)-Indoprofe

    Others Inflammation/Immunology
    Indoprofen is a non-steroidal anti-inflammatory drug, provide insight into treatments for spinal muscular atrophies.
  • HY-101792
    RG7800

    RO6885247

    DNA/RNA Synthesis Others
    RG7800 is a SMN2 splicing modifier. RG7800 has the potential for spinal muscular atrophy treatment.
  • HY-112633
    SMN-C3

    DNA/RNA Synthesis Others
    SMN-C3 is an orally active SMN2 splicing modulator and has the potential to treat spinal muscular atrophy (SMA).
  • HY-101792A
    RG7800 hydrochloride

    RO6885247 hydrochloride

    DNA/RNA Synthesis Neurological Disease
    RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy.
  • HY-112980
    Nusinersen

    DNA/RNA Synthesis Inflammation/Immunology
    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.
  • HY-111055
    BIP-135

    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.
  • HY-120508
    Pivanex

    AN-9; Pivalyloxymethyl butyrate

    HDAC Bcr-Abl Apoptosis Cancer Inflammation/Immunology
    Pivanex (AN-9), a derivative of Butyric acid, is an orally active HDAC inhibitor. Pivanex down-regulates bcr-abl protein and enhances apoptosis. Pivanex has antimetastic and antiangiogenic properties.