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Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!2025-02-06
15 Results for "
atrophy,SMA
" in MedChemExpress (MCE) Product Catalog:
Products (15)
15 Results for "atrophy,SMA" in MCE Product Catalog:
16
Target:
Domaines de recherche:
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 .
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16 Cited Publications
Cat. No.: HY-19620A
CAS No.: 1562338-39-9
Synonyms: LMI070 hydrochloride; NVS-SM1 hydrochloride
Target:
Domaines de recherche:
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 .
Cat. No.: HY-P99518
CAS No.: 1580555-26-5
Synonyms: BMS 986089; RG 6206; RO 7239361; BHV2000
Target:
Domaines de recherche:
Cat. No.: HY-176803
CAS No.: 2673403-86-4
Target:
Domaines de recherche:
MF-DH-300 is a 15-PGDH inhibitor that can be applicable to the research of muscle disorders such as spinal muscular atrophy (SMA).
Target:
Domaines de recherche:
SMN-C3 is an orally active SMN2 splicing modulator and has the potential to treat spinal muscular atrophy (SMA).
Target:
Domaines de recherche:
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 .
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Domaines de recherche:
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 .
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PF-DcpSi is a mRNA decapping scavenger enzyme (DcpS) inhibitor (IC50: 0.11 nM). PF-DcpSi (30 mg/kg,i.p.) ameliorates the disease phenotype in a mice model of spinal muscular atrophy (SMA) .
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Domaines de recherche:
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 .
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RNA, [2'-O-(2-methylamino)-2-oxoethyl](m5C-sp-A-m5C-sp-m5U-m5U-sp-m5U-sp-m5C-sp-A-sp-m5U-sp-A-sp-A-sp-m5U-sp-G-sp-m5C-sp-m5U-sp-G-sp-G-sp-m5C)
Cat. No.: HY-177661
CAS No.: 2701584-16-7
Synonyms: BIIB115; ION306
Cat. No.: HY-117000A
Domaines de recherche:
D156844 (Compound 11a) hydrochloride is a SMN2 promoter activator with an EC50 of 4 nM. D156844 hydrochloride increases the mRNA expression of the mouse SMN in NSC-34 cells and human SMN2 promoter in severe type I spinal muscular atrophy (SMA) fibroblasts as well as full-length human SMN protein. D156844 hydrochloride overcomes DHFR inhibition. D156844 hydrochloride can be used for SMA research .
Cat. No.: HY-119460
CAS No.: 337932-29-3
Target:
Domaines de recherche:
Cuspin-1 is a upregulator of Survival of Motor Neuron protein (SMN). Cuspin-1 upregulates SMN expression post-transcriptionally, and increases the phosphorylation of Erk. Cuspin-1 can be used for research of neurodegenerative disease, such as spinal muscular atrophy (SMA) .
Cat. No.: HY-117000
CAS No.: 1005501-84-7
Domaines de recherche:
D156844 (Compound 11a) is a SMN2 promoter activator with an EC50 of 4 nM. D156844 increases the mRNA expression of the mouse SMN in NSC-34 cells and human SMN2 promoter in severe type I spinal muscular atrophy (SMA) fibroblasts as well as full-length human SMN protein. D156844 overcomes DHFR inhibition. D156844 can be used for SMA research .
0 Images
RNA, [2'-O-(2-methylamino)-2-oxoethyl](m5C-sp-A-m5C-sp-m5U-m5U-sp-m5U-sp-m5C-sp-A-sp-m5U-sp-A-sp-A-sp-m5U-sp-G-sp-m5C-sp-m5U-sp-G-sp-G-sp-m5C), sodium salt
Cat. No.: HY-177661A
Synonyms: BIIB115 sodium; ION306 sodium
Cat. No.: HY-L086
3,671 compounds
Neurodegenerative diseases are incurable and life-threatening conditions that result in progressive degeneration and/or death of nerve cells. Some common neurodegenerative diseases include Alzheimer’s Disease (AD), Parkinson’s Disease (PD), Motor Neuron Disease (MND), Huntington’s Disease (HD), Spino-Cerebellar Ataxia (SCA), Spinal Muscular Atrophy (SMA), and Amyotrophic Lateral Sclerosis (ALS). Because the pathophysiology of neurodegenerative disorders is generally poorly understood, it is difficult to identify promising molecular targets and validate them. At the same time, about 85% of the drugs fail in clinical trials. Therefore, validating new targets and discovering new drugs to mitigate neurodegenerative disorders is need of the hour.
MCE offers a unique collection of 3,671 compounds with anti-Neurodegenerative Diseases activities or targeting the unique targets of neurodegenerative diseases. MCE Neurodegenerative Disease-related Compound Library is a useful tool for exploring the mechanism of neurodegenerative diseases and discovering new drugs for neurodegenerative diseases.
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Protocols
Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!A suitable model is crucial in drug screening experiments. Organs can mimic the three-dimensional functional structure of internal organs, have similar spatial organization to corresponding organs, maintain some key characteristics, and reproduce some physiological functions. They are widely used for modeling and personalized drug screening physiological functions.2025-02-06
