spinal muscular atrophy

By Targets:	Apoptosis (1) Bcr-Abl (1) DNA/RNA Synthesis (10) GSK-3 (1) HDAC (1) Potassium Channel (2)
By Research Areas:	Cancer (3) Inflammation/Immunology (5) Neurological Disease (5)

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

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-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 .

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 .

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

5 Publications Verification

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).

Indoprofen (±)-Indoprofe	Others	Inflammation/Immunology
Indoprofen is a non-steroidal anti-inflammatory drug, provide insight into treatments for spinal muscular atrophies.

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

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-112980

Nusinersen	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 .

HY-112980A

Nusinersen sodium	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 .

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-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-120508

Pivanex AN-9; Pivalyloxymethyl butyrate	HDAC Bcr-Abl Apoptosis	Inflammation/Immunology Cancer
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 .

Cat. No.	Product Name

HY-L086

Neurodegenerative Disease-related Compound Library	2228 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 2228 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

2228 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 2228 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-P99441

Apitegromab SRK-015	Inhibitory Antibodies	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	Inhibitory Antibodies	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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