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Results for "

atrophy,SMA

" in MedChemExpress (MCE) Product Catalog:

By Targets:	DNA/RNA Synthesis (7) ERK (1) Potassium Channel (2) TGF-beta/Smad (1)
By Research Areas:	Cancer (2) Inflammation/Immunology (1) Neurological Disease (4)

Inhibitors & Agonists

Screening Libraries

Inhibitory Antibodies

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

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-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-P99518

Taldefgrobep alfa BMS 986089; RG 6206; RO 7239361; BHV2000	TGF-beta/Smad	Others
Taldefgrobep alfa (BMS 986089; RG 6206; RO 7239361) is a potent inhibitory antibody targeting to human 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-19620A

Branaplam hydrochloride 10+ Cited Publications 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 10+ Cited Publications 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-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-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) .

Cat. No.	Product Name

HY-L086

Neurodegenerative Disease-related Compound Library	2,733 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 2,733 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

2,733 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 2,733 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

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 potent inhibitory antibody targeting to human 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 .

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