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Results for "

spinal+muscular+atrophy

" in MedChemExpress (MCE) Product Catalog:

By Targets:	15-PGDH (1) Dihydrofolate reductase (DHFR) (2) DNA/RNA Synthesis (18) ERK (1) Potassium Channel (2) Reference Standards (2) TGF-beta/Smad (3)
By Research Areas:	Cancer (2) Inflammation/Immunology (2) Neurological Disease (15)
Oligonucleotides:	Antisense Oligonucleotides (4)

Inhibitors & Agonists

Screening Libraries

Inhibitory Antibodies

Oligonucleotides

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

HY-112980

Nusinersen 4 Publications Verification	DNA/RNA Synthesis	Neurological Disease
Nusinersen is an antisense oligonucleotide active molecule. Nusinersen modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting the production of full-length SMN protein. Nusinersen improves spinal muscular atrophy .

HY-19620

Branaplam Maximum Cited Publications 15 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 .

HY-P99441

Apitegromab SRK-015	TGF-beta/Smad	Neurological Disease
Apitegromab (SRK-015) is an anti-promyostatin monoclonal antibody. Apitegromab can be used for the research of neuromuscular disease including spinal muscular atrophy .

HY-112980A

Nusinersen sodium 4 Publications Verification	DNA/RNA Synthesis	Neurological Disease
Nusinersen sodium is an antisense oligonucleotide active molecule. Nusinersen sodium modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting the production of full-length SMN protein. Nusinersen sodium improves spinal muscular atrophy .

HY-102020

RG3039 3 Publications Verification PF-06687859	DNA/RNA Synthesis	Neurological Disease
RG3039 (PF-06687859) is an orally active, blood-brain barrier-permeable DcpS inhibitor with an IC₅₀ of 4.2 nM against hDcpS. RG3039 inhibits the decapping activity of DcpS and maintains DcpS in a catalytically inactive conformation in the central nervous system and other tissues. RG3039 extends survival and improves function in spinal muscular atrophy mice. RG3039 is applicable to research related to spinal muscular atrophy .

HY-P99518

Taldefgrobep alfa BMS 986089; RG 6206; RO 7239361; BHV2000	TGF-beta/Smad	Others
Taldefgrobep alfa (BMS 986089; RG 6206; RO 7239361) is a protein that blocks myostatin. Taldefgrobep alfa is a fusion protein composed of a human IgG1-Fc domain and Adnectin domain. Taldefgrobep alfa can be used for spinal muscular atrophy (SMA) research .

HY-176803

MF-DH-300	15-PGDH	Neurological Disease
MF-DH-300 is a 15-PGDH inhibitor that can be applicable to the research of muscle disorders such as spinal muscular atrophy (SMA).

HY-19620A

Branaplam hydrochloride Maximum Cited Publications 15 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 .

HY-101792

RG7800 5 Publications Verification RO6885247	DNA/RNA Synthesis	Others
RG7800 is a SMN2 splicing modifier. RG7800 has the potential for spinal muscular atrophy treatment.

HY-P990993

Emugrobart GYM-329, RG6237, RG-70240	TGF-beta/Smad	Inflammation/Immunology
Emugrobart (GYM-329, RG6237, RG-70240) is a humanized IgG1κ antibody targeting myostatin (Myostatin; GDF8). Emugrobart binds to pro-myostatin and latent myostatin, blocking their cleavage into mature myostatin; it also has a clearance function, which transports bound myostatin for degradation and allows for cyclic reuse. Emugrobart enhances muscle strength in mouse models of muscle atrophy and increases muscle mass in cynomolgus monkeys. Emugrobart can be used in research on spinal muscular atrophy and facioscapulohumeral muscular dystrophy .

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 tetrahydrochloride 5 Publications Verification RO6885247 tetrahydrochloride	DNA/RNA Synthesis	Neurological Disease
RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC_1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy.

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-124648

SMN-C2	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 .

HY-122604

PF-DcpSi	DNA/RNA Synthesis	Neurological Disease
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) .

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-117000A

D156844 hydrochloride	DNA/RNA Synthesis Dihydrofolate reductase (DHFR)	Neurological Disease
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 .

HY-119460

Cuspin-1	ERK	Neurological Disease
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) .

HY-117000

D156844	DNA/RNA Synthesis Dihydrofolate reductase (DHFR)	Neurological Disease
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 .

HY-102020A

RG3039 hydrochloride	DNA/RNA Synthesis	Neurological Disease
RG3039 hydrochloride is an orally active, blood-brain barrier-permeable DcpS inhibitor with an IC₅₀ of 4.2 nM against hDcpS. RG3039 hydrochloride inhibits the decapping activity of DcpS and maintains DcpS in a catalytically inactive conformation in the central nervous system and other tissues. RG3039 hydrochloride extends survival and improves function in spinal muscular atrophy mice. RG3039 hydrochloride is applicable to research related to spinal muscular atrophy .

HY-102020R

RG3039 (Standard) PF-06687859 (Standard)	DNA/RNA Synthesis Reference Standards	Neurological Disease
RG3039 (Standard) is the analytical standard of RG3039 (HY-102020). This product is intended for research and analytical applications. RG3039 (PF-06687859) is an orally active, blood-brain barrier-permeable DcpS inhibitor with an IC₅₀ of 4.2 nM against hDcpS. RG3039 inhibits the decapping activity of DcpS and maintains DcpS in a catalytically inactive conformation in the central nervous system and other tissues. RG3039 extends survival and improves function in spinal muscular atrophy mice. RG3039 is applicable to research related to spinal muscular atrophy .

HY-101792R

RG7800 (Standard) RO6885247 (Standard)	Reference Standards DNA/RNA Synthesis	Others
RG7800 (Standard) (RO6885247 (Standard)) is the analytical standard of RG7800 (HY-101792). This product is intended for research and analytical applications. RG7800 is a SMN2 splicing modifier. RG7800 has the potential for spinal muscular atrophy treatment.

HY-124640

SMN2 modulator-1	DNA/RNA Synthesis	Neurological Disease
SMN2 modulator-1 is a brain-penetrant survival motor neuron (SMN) modulator. SMN2 modulator-1 post-translationally stabilizes SMN protein and increases SMN protein levels independent of SMN2 transcription. SMN2 modulator-1 can be used for the research of spinal muscular atrophy^[1].

Cat. No.	Product Name

HY-L086

Neurodegenerative Disease-related Compound Library	3,545 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 3,545 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.

Neurodegenerative Disease-related Compound Library

3,545 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 3,545 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.

Cat. No.	Product Name	Target	Research Area	Image

HY-P99441

Apitegromab SRK-015	TGF-beta/Smad	Neurological Disease
Apitegromab (SRK-015) is an anti-promyostatin monoclonal antibody. Apitegromab can be used for the research of neuromuscular disease including spinal muscular atrophy .

HY-P99518

Taldefgrobep alfa BMS 986089; RG 6206; RO 7239361; BHV2000	TGF-beta/Smad	Others
Taldefgrobep alfa (BMS 986089; RG 6206; RO 7239361) is a protein that blocks myostatin. Taldefgrobep alfa is a fusion protein composed of a human IgG1-Fc domain and Adnectin domain. Taldefgrobep alfa can be used for spinal muscular atrophy (SMA) research .

HY-P990993

Emugrobart GYM-329, RG6237, RG-70240	TGF-beta/Smad	Inflammation/Immunology
Emugrobart (GYM-329, RG6237, RG-70240) is a humanized IgG1κ antibody targeting myostatin (Myostatin; GDF8). Emugrobart binds to pro-myostatin and latent myostatin, blocking their cleavage into mature myostatin; it also has a clearance function, which transports bound myostatin for degradation and allows for cyclic reuse. Emugrobart enhances muscle strength in mouse models of muscle atrophy and increases muscle mass in cynomolgus monkeys. Emugrobart can be used in research on spinal muscular atrophy and facioscapulohumeral muscular dystrophy .

Cat. No.	Product Name		Classification

HY-112980

Nusinersen 4 Publications Verification		Antisense Oligonucleotides
Nusinersen is an antisense oligonucleotide active molecule. Nusinersen modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting the production of full-length SMN protein. Nusinersen improves spinal muscular atrophy .

HY-112980A

Nusinersen sodium 4 Publications Verification		Antisense Oligonucleotides
Nusinersen sodium is an antisense oligonucleotide active molecule. Nusinersen sodium modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting the production of full-length SMN protein. Nusinersen sodium improves spinal muscular atrophy .

HY-177661

Salanersen BIIB115; ION306		Antisense Oligonucleotides
Salanersen is an antisense oligonucleotide targeted to survival motor neuron 2 (SMN2). It is used for the study of spinal muscular atrophy (SMA).

HY-177661A

Salanersen sodium BIIB115 sodium; ION306 sodium		Antisense Oligonucleotides
Salanersen sodium is an antisense oligonucleotide targeted to survival motor neuron 2 (SMN2). It is used for the study of spinal muscular atrophy (SMA).

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