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Results for "

Duchenne muscular dystrophy

" in MedChemExpress (MCE) Product Catalog:

By Targets:	Apoptosis (2) Arp2/3 Complex (4) Cannabinoid Receptor (1) CDK (2) Cytochrome P450 (1) DNA/RNA Synthesis (3) Drug Derivative (1) Endogenous Metabolite (3) Glucocorticoid Receptor (2) Guanylate Cyclase (1) HDAC (2) Liposome (1) Microtubule/Tubulin (2) Mineralocorticoid Receptor (1) Myosin (1) NF-κB (2) Nucleoside Antimetabolite/Analog (2) PPAR (1) REV-ERB (1) TGF-beta/Smad (2)
By Research Areas:	Cancer (5) Cardiovascular Disease (1) Infection (1) Inflammation/Immunology (5) Metabolic Disease (14) Neurological Disease (3)
Oligonucleotides:	Nucleotides and their Analogs (1) Cationic Lipids (1) Antisense Oligonucleotides (14)

Inhibitors & Agonists

Biochemical Assay Reagents

Inhibitory Antibodies

Natural
Products

Oligonucleotides

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

HY-101459

RTC13	Drug Derivative	Metabolic Disease
RTC13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy (DMD) .

HY-108753

Eteplirsen AVI 4658	Arp2/3 Complex	Metabolic Disease
Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research .

HY-122631

TG693	CDK	Others
TG693 is an orally active inhibitor of CLK1. TG693 regulates the mutated exon 31 of the dystrophin gene in vivo. TG693 is used in Duchenne muscular dystrophy (DMD) research .

HY-P99857

Domagrozumab PF-06252616	TGF-beta/Smad	Metabolic Disease
Domagrozumab is an anti-myostatin humanized monoclonal antibody with a K_D value of 2.6 pM for human myostatin. Domagrozumab induces muscle anabolic activity. Domagrozumab can be used in research of duchenne muscular dystrophy (DMD) .

HY-127137

Adenylosuccinic acid Adenylosuccinate; Aspartyl adenylate	Endogenous Metabolite	Metabolic Disease
Adenylosuccinic acid (Adenylosuccinate; Aspartyl adenylate) is a purine ribonucleoside monophosphate and plays a role in nucleotide cycle metabolite. Adenylosuccinic acid can be converted into fumaric acid through adenylosuccinate lyase. Adenylosuccinic acid has the potential for the study of duchenne muscular dystrophy(DMD) .

HY-127137A

Adenylosuccinic acid tetraammonium Adenylosuccinate tetraammonium; Aspartyl adenylate tetraammonium	Endogenous Metabolite	Metabolic Disease
Adenylosuccinic acid tetraammonium (Adenylosuccinate; Aspartyl adenylate) is an orally active purine ribonucleoside monophosphate and plays a role in nucleotide cycle metabolite. Adenylosuccinic acid tetraammonium can be converted into fumaric acid through adenylosuccinate lyase. Adenylosuccinic acid tetraammonium has the potential for the study of duchenne muscular dystrophy(DMD) .

HY-134377

Bocidelpar ASP0367; MA-0211	PPAR	Metabolic Disease
Bocidelpar is a modulator of peroxisome proliferator-activated receptor delta (PPAR-δ). Bocidelpar improves mitochondrial biogenesis and function in Duchenne Muscular Dystrophy (DMD) muscle cells .

HY-161050

YSR734	HDAC Apoptosis	Cancer
YSR734 (Compound 21) is a covalent HDAC inhibitor with IC₅₀ values of 110 nM, 154 nM, and 143 nM for HDAC1, HDAC2, and HDAC3, respectively. YSR734 can induce apoptosis in leukemia cells. YSR734 can induce myoblast differentiation and is used in the study of Duchenne muscular dystrophy .

HY-17614

Ezutromid SMT C1100; BMN 195; VOX-C1100	Cytochrome P450	Others
Ezutromid (SMT C1100) is a first-in-class, orally active benzoxazole utrophin modulator with an EC₅₀ of 0.91 μM. Ezutromid can be used for the research Duchenne muscular dystrophy (DMD). Ezutromid inhibits CYP1A2 enzymic activity in human liver microsomes (HLM) with an IC₅₀ of 5.4 μM .

HY-132611

Golodirsen SRP-4053	Arp2/3 Complex	Others
Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) that specifically targets exon 53 of dystrophin pre-mRNA. Golodirsen can be used for the research of Duchenne muscular dystrophy (DMD) .

HY-14415

SR8278 5+ Cited Publications	REV-ERB	Neurological Disease Metabolic Disease
SR8278 is a REV-ERBα antagonist and inhibits the REV-ERBα transcriptional repression activity with an EC₅₀ of 0.47 μM. SR8278 is used to regulate the metabolism in organisms and study biological rhythm. SR8278 also can be used for the research of Duchenne muscular dystrophy and Alzheimer's disease .

HY-132586

Viltolarsen NS-065/NCNP-01	Nucleoside Antimetabolite/Analog	Metabolic Disease
Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research .

HY-132586A

Viltolarsen sodium NS-065/NCNP-01 sodium	Nucleoside Antimetabolite/Analog	Metabolic Disease
Viltolarsen (NS-065/NCNP-01) sodium is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen sodium binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen sodium has the potential for Duchenne muscular dystrophy (DMD) research .

HY-14842

Givinostat Maximum Cited Publications 11 Publications Verification ITF-2357	HDAC	Inflammation/Immunology Cancer
Givinostat (ITF-2357) is a HDAC inhibitor with an IC₅₀ of 198 and 157 nM for HDAC1 and HDAC3, respectively. Givinostat can be used for Duchenne muscular dystrophy (DMD) research .

HY-W272699

Utrophin activator-1	Others	Metabolic Disease
Utrophin activator-1 (compound 3) is an utrophin upregulator with an EC₅₀ of 1.8 μM. Utrophin activator-1 can be used for Duchenne muscular dystrophy research .

HY-145724

Drisapersen Kyndrisa; GSK2402968A; PRO051	DNA/RNA Synthesis	Others
Drisapersen, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.

HY-150078

OX01914	Microtubule/Tubulin	Others
OX01914 is a water-solube and permeable utrophin modulator (upregulates utrophin protein levels), with an EC₅₀ of 20.5 μM. OX01914 can be used in study of duchenne muscular dystrophy (DMD) .

HY-150077

Utrophin modulator 1	Microtubule/Tubulin	Others
Utrophin modulator 1 is a potent utrophin modulator (upregulates utrophin protein levels), with an EC₅₀ of 0.11 μM. Utrophin modulator 1 can be used in study of duchenne muscular dystrophy (DMD) .

HY-150237

FITC-labeled Drisapersen sodium	DNA/RNA Synthesis	Others
FITC-labeled Drisapersen (sodium) is Drisapersen labeled with FITC. Drisapersen, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.

HY-138121

PSB-KD107	Cannabinoid Receptor	Inflammation/Immunology
PSB-KD107 is an agonist of the cannabinoid activated orphan G-protein-coupled receptor GPR18, and PSB-KD107 has anti-inflammatory activity. PSB-KD107 can be used in the study of Duchenne muscular dystrophy .

HY-132584A

Casimersen sodium SRP-4045 sodium	Arp2/3 Complex	Others
Casimersen sodium is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass. Casimersen sodium binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame (by skipping exon 45) resulting in the production of an internally truncated but functional dystrophin protein. Casimersen sodium can be used for the research of Duchenne muscular dystrophy (DMD) .

HY-W015828

Ompenaclid 2 Publications Verification RGX-202; 3-Guanidinopropionic acid	Apoptosis Endogenous Metabolite	Cancer
Ompenaclid (RGX-202) is an oral small-molecule SLC6A8 transporter inhibitor. Ompenaclid robustly inhibits creatine import in vitro and in vivo, reduces intracellular phosphocreatine and ATP levels, and induces tumor apoptosis. Ompenaclid can be used for the research of cancer and duchenne muscular dystrophy .

HY-132584

Casimersen SRP-4045	Arp2/3 Complex	Others
Casimersen (SRP-4045) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass. Casimersen binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame (by skipping exon 45) resulting in the production of an internally truncated but functional dystrophin protein. Casimersen can be used for the research of Duchenne muscular dystrophy (DMD) .

HY-152219

CLK1-IN-2	CDK	Infection Cancer
CLK1-IN-2 is metabolically stable Clk1 inhibitor. CLK1-IN-2 has selectivity for Clk1 with an IC₅₀ value of 1.7 nM. CLK1-IN-2 can be used for the research of tumour, Duchenne's muscular dystrophy and viral infections such as HIV-1 and influenza .

HY-148799

Sevasemten	Myosin	Others
Sevasemten is an orally active allosteric inhibitor of skeletal muscle myosin that protects skeletal muscle from contraction-induced injury. Sevasemten exhibits selectively myosin inhibition with IC₅₀s of ≤10 μM (skeletal), >100 μM (cardiac), respectively. Sevasemten decreases muscle damage biomarkers and fibrosis while increasing muscle strength and activity in in Duchenne muscular dystrophy disease models .

HY-153369

BAY-747	Guanylate Cyclase	Cardiovascular Disease Neurological Disease
BAY-747 is an orally active and brain-penetrant stimulator of soluble guanylate cyclase (sGC). BAY-747 reverses L-NAME induced memory impairments and enhances cognition of rats in the object location task (OLT). BAY-747 also decreases blood pressure in both conscious normotensive and spontaneously hypertensive rats (SHR). BAY-747 improves function of the skeletal muscle associated with Duchenne muscular dystrophy (DMD) in mdx/mTRG2 mice model .

HY-109017

Vamorolone VBP15	Glucocorticoid Receptor Mineralocorticoid Receptor NF-κB	Inflammation/Immunology
Vamorolone (VBP15) is a first-in-class, orally active dissociative steroidal anti-inflammatory agent and membrane-stabilizer. Vamorolone improves muscular dystrophy without side effects. Vamorolone shows potent NF-κB inhibition and substantially reduces hormonal effects .

HY-123359

RTC14	DNA/RNA Synthesis	Others
RTC14 is a read-through compound (RTC) that can induce ribosomes to bypass nonsense mutations in mRNA and allow the production of full-length functional proteins. RTC14 has the potential to be used in the research of various genetic disorders, such as nonsense mutations in the ataxia-telangiectasia mutated (ATM) gene and the dystrophin gene .

HY-136340

21-Acetoxypregna-1,4,9(11),16-tetraene-3,20-dione	Glucocorticoid Receptor NF-κB	Metabolic Disease Inflammation/Immunology
21-Acetoxypregna-1,4,9(11),16-tetraene-3,20-dione is an intermediate of delta 9,11 steroids synthesis, for example, Vamorolone (HY-109017). The delta 9,11 steroids are modifications of glucocorticoids and has anti-inflammatory properties. The delta 9,11 steroids are agents for protection against cell damage (lipid peroxidation) and inhibition of neovascularization .

HY-P99370

Landogrozumab LY2495655; Anti-GDF8 / Myostatin Reference Antibody (landogrozumab)	TGF-beta/Smad	Inflammation/Immunology Cancer
Landogrozumab (LY2495655) is an humanized anti-myostatin monoclonal antibody. Landogrozumab effectively improves muscle volume, hand grip strength and function. Landogrozumab can be used for the research of muscle wasting disease .

HY-147332

TCL053	Liposome	Neurological Disease
TCL053 is an ionizable lipid carrier and used to introduce active components, in particular nucleic acids, into cells with excellent efriciency. TCL053, together with DPPC (Dipalmitoylphosphatidylcholine), PEG-DMG (Polyethylene glycoldimyristoyl glycerol), and cholesterol, forms lipid nanoparticle (LNP) which is able to deliver Cas9 mRNA and sgRNA into skeletal muscle .

Cat. No.	Product Name	Type

HY-147332

TCL053	Drug Delivery
TCL053 is an ionizable lipid carrier and used to introduce active components, in particular nucleic acids, into cells with excellent efriciency. TCL053, together with DPPC (Dipalmitoylphosphatidylcholine), PEG-DMG (Polyethylene glycoldimyristoyl glycerol), and cholesterol, forms lipid nanoparticle (LNP) which is able to deliver Cas9 mRNA and sgRNA into skeletal muscle .

Cat. No.	Product Name	Target	Research Area

HY-P99857

Domagrozumab PF-06252616	TGF-beta/Smad	Metabolic Disease
Domagrozumab is an anti-myostatin humanized monoclonal antibody with a K_D value of 2.6 pM for human myostatin. Domagrozumab induces muscle anabolic activity. Domagrozumab can be used in research of duchenne muscular dystrophy (DMD) .

HY-P99370

Landogrozumab LY2495655; Anti-GDF8 / Myostatin Reference Antibody (landogrozumab)	TGF-beta/Smad	Inflammation/Immunology Cancer
Landogrozumab (LY2495655) is an humanized anti-myostatin monoclonal antibody. Landogrozumab effectively improves muscle volume, hand grip strength and function. Landogrozumab can be used for the research of muscle wasting disease .

Adenylosuccinic acid Adenylosuccinate; Aspartyl adenylate	Human Gut Microbiota Metabolites Microorganisms Classification of Application Fields Source classification Metabolic Disease Endogenous metabolite Disease Research Fields	Endogenous Metabolite
Adenylosuccinic acid (Adenylosuccinate; Aspartyl adenylate) is a purine ribonucleoside monophosphate and plays a role in nucleotide cycle metabolite. Adenylosuccinic acid can be converted into fumaric acid through adenylosuccinate lyase. Adenylosuccinic acid has the potential for the study of duchenne muscular dystrophy(DMD) .

Ompenaclid 2 Publications Verification RGX-202; 3-Guanidinopropionic acid	Structural Classification Natural Products Classification of Application Fields Source classification Endogenous metabolite Disease Research Fields Cancer	Apoptosis Endogenous Metabolite
Ompenaclid (RGX-202) is an oral small-molecule SLC6A8 transporter inhibitor. Ompenaclid robustly inhibits creatine import in vitro and in vivo, reduces intracellular phosphocreatine and ATP levels, and induces tumor apoptosis. Ompenaclid can be used for the research of cancer and duchenne muscular dystrophy .

Cat. No.	Product Name		Classification

HY-108753A

Eteplirsen sodium AVI 4658 sodium		Antisense Oligonucleotides
Eteplirsen (AVI 4658) sodium is a synthetic antisense oligonucleotide that induces dystrophin production. Eteplirsen (AVI 4658) sodium promotes exon 51 skipping in Duchenne muscular dystrophy patients and can be used in Duchenne muscular dystrophy research .

HY-108753

Eteplirsen AVI 4658		Antisense Oligonucleotides
Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research .

HY-132611A

Golodirsen sodium SRP-4053 sodium		Antisense Oligonucleotides
Golodirsen sodium is a phosphorodiamidate morpholino oligomer (PMO) that specifically targets exon 53 of dystrophin pre-mRNA. Golodirsen sodium can be used for the research of Duchenne muscular dystrophy (DMD) .

HY-127137A

Adenylosuccinic acid tetraammonium Adenylosuccinate tetraammonium; Aspartyl adenylate tetraammonium		Nucleotides and their Analogs
Adenylosuccinic acid tetraammonium (Adenylosuccinate; Aspartyl adenylate) is an orally active purine ribonucleoside monophosphate and plays a role in nucleotide cycle metabolite. Adenylosuccinic acid tetraammonium can be converted into fumaric acid through adenylosuccinate lyase. Adenylosuccinic acid tetraammonium has the potential for the study of duchenne muscular dystrophy(DMD) .

HY-132586A

Viltolarsen sodium NS-065/NCNP-01 sodium		Antisense Oligonucleotides
Viltolarsen (NS-065/NCNP-01) sodium is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen sodium binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen sodium has the potential for Duchenne muscular dystrophy (DMD) research .

HY-132592A

Suvodirsen sodium WVE-210201 sodium		Antisense Oligonucleotides
Suvodirsen sodium induces exon 51 skipping and has the potential for study Duchenne muscular dystrophy (DMD) .

HY-132592

Suvodirsen WVE-210201		Antisense Oligonucleotides
Suvodirsen (WVE-210201) is a oligonucleotide. Suvodirsen has the potential for study Duchenne muscular dystrophy (DMD) .

HY-132585A

SRP-5051 sodium Vesleteplirsen sodium		Antisense Oligonucleotides
SRP-5051 sodium is a next-generation antisense oligonucleotide of peptide phosphorodiamidate morpholino oligomer (PPMO). SRP-5051 targeting exon 51 skipping in Duchenne muscular dystrophy (DMD) .

HY-132611

Golodirsen SRP-4053		Antisense Oligonucleotides
Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) that specifically targets exon 53 of dystrophin pre-mRNA. Golodirsen can be used for the research of Duchenne muscular dystrophy (DMD) .

HY-132586

Viltolarsen NS-065/NCNP-01		Antisense Oligonucleotides
Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research .

HY-145724A

Drisapersen sodium Kyndrisa sodium; GSK2402968A sodium; PRO051 sodium		Antisense Oligonucleotides
Drisapersen sodium, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.

HY-145724

Drisapersen Kyndrisa; GSK2402968A; PRO051		Antisense Oligonucleotides
Drisapersen, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.

HY-150237

FITC-labeled Drisapersen sodium		Antisense Oligonucleotides
FITC-labeled Drisapersen (sodium) is Drisapersen labeled with FITC. Drisapersen, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.

HY-132584A

Casimersen sodium SRP-4045 sodium		Antisense Oligonucleotides
Casimersen sodium is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass. Casimersen sodium binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame (by skipping exon 45) resulting in the production of an internally truncated but functional dystrophin protein. Casimersen sodium can be used for the research of Duchenne muscular dystrophy (DMD) .

HY-132584

Casimersen SRP-4045		Antisense Oligonucleotides
Casimersen (SRP-4045) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass. Casimersen binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame (by skipping exon 45) resulting in the production of an internally truncated but functional dystrophin protein. Casimersen can be used for the research of Duchenne muscular dystrophy (DMD) .

HY-147332

TCL053		Cationic Lipids
TCL053 is an ionizable lipid carrier and used to introduce active components, in particular nucleic acids, into cells with excellent efriciency. TCL053, together with DPPC (Dipalmitoylphosphatidylcholine), PEG-DMG (Polyethylene glycoldimyristoyl glycerol), and cholesterol, forms lipid nanoparticle (LNP) which is able to deliver Cas9 mRNA and sgRNA into skeletal muscle .

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Duchenne muscular dystrophy

Inhibitors & Agonists

Biochemical Assay Reagents

Inhibitory Antibodies

NaturalProducts

Oligonucleotides

Natural
Products