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Results for "

Duchenne muscular dystrophy (DMD)

" in MedChemExpress (MCE) Product Catalog:

By Targets:	Antibiotic (1) CDK (1) Cytochrome P450 (2) DNA/RNA Synthesis (4) Dystrophin (11) Endogenous Metabolite (2) Guanylate Cyclase (1) HDAC (2) Microtubule/Tubulin (2) Nucleoside Antimetabolite/Analog (2) PPAR (1) TGF-beta/Smad (1)
By Research Areas:	Cancer (2) Cardiovascular Disease (2) Inflammation/Immunology (2) Metabolic Disease (10) Neurological Disease (3)
Oligonucleotides:	Nucleotide Analogs (1) Antisense Oligonucleotides (12)

Inhibitors & Agonists

Inhibitory Antibodies

Natural
Products

Oligonucleotides

Targets Recommended: Cdc42-binding kinase

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

HY-132611

Golodirsen SRP-4053	Dystrophin	Neurological Disease
Golodirsen (SRP-4053) is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen can restore the expression of the anti-myostatin protein. Golodirsen can be used for the research of duchenne muscular dystrophy (DMD) .

HY-132611A

Golodirsen sodium SRP-4053 sodium	DNA/RNA Synthesis Dystrophin	Neurological Disease
Golodirsen (SRP-4053) sodium is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen sodium restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen sodium can restore the expression of the anti-myostatin protein. Golodirsen sodium can be used for the research of duchenne muscular dystrophy (DMD) .

HY-101459

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

HY-14842

Givinostat 10+ Cited Publications 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-145724

Drisapersen Kyndrisa; GSK2402968A; PRO051	DNA/RNA Synthesis Dystrophin	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-122631

TG693	CDK Dystrophin	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-177659

Rimigorsen	Dystrophin	Others
Rimigorsen is an antisense oligonucleotide that induces skipping of exon 44 of the pre-mRNA encoding dystrophin in a Duchenne muscular dystrophy (DMD)

HY-177659A

Rimigorsen sodium	Dystrophin	Others
Rimigorsen sodium is an antisense oligonucleotide that induces skipping of exon 44 of the pre-mRNA encoding dystrophin in a Duchenne muscular dystrophy (DMD)

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-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-122588A

Negamycin hydrochloride	Antibiotic	Cardiovascular Disease Metabolic Disease
Negamycin (hydrochloride) is a dipeptide antibiotic. Negamycin (hydrochloride) can restore dystrophin expression in skeletal and cardiac muscles in mdx Duchenne muscular dystrophy (DMD) murine model. Negamycin (hydrochloride) can bind to a partial sequence of the eukaryotic rRNA-decoding A-site. Negamycin (hydrochloride) can be studied in DMD research .

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-150237

FITC-labeled Drisapersen sodium	DNA/RNA Synthesis Dystrophin	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-14842R

Givinostat (Standard)	HDAC	Inflammation/Immunology Cancer
Givinostat (Standard) is the analytical standard of Givinostat. This product is intended for research and analytical applications. 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-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-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-132586A

Viltolarsen sodium NS-065/NCNP-01 sodium	Nucleoside Antimetabolite/Analog Dystrophin	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-132586

Viltolarsen NS-065/NCNP-01	Nucleoside Antimetabolite/Analog Dystrophin	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-17614R

Ezutromid (Standard)	Cytochrome P450	Others
Ezutromid (Standard) is the analytical standard of Ezutromid. This product is intended for research and analytical applications. 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-123359

RTC14	DNA/RNA Synthesis Dystrophin	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-134377

Bocidelpar ASP0367; MA-0211	PPAR	Metabolic Disease
Bocidelpar (ASP0367; MA-0211) is a selective, orally active PPARδ modulator. Bocidelpar activates the PPARδ downstream signaling pathway, upregulates the expression of target genes such as ABCA1 and ACAA2. Bocidelpar then promotes fatty acid oxidation (FAO) and mitochondrial biogenesis, and improves mitochondrial dysfunction. Bocidelpar can improve mitochondrial biogenesis and function in muscle cells. Bocidelpar is mainly used in the study of mitochondrial dysfunction diseases such as primary mitochondrial myopathy (PMM) and Duchenne muscular dystrophy (DMD) .

HY-153369

BAY-747	Guanylate Cyclase	Cardiovascular Disease Neurological Disease
BAY-747 (BAY 1165747) 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 .

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) .

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) .

Cat. No.	Product Name		Classification

HY-132611

Golodirsen SRP-4053		Antisense Oligonucleotides
Golodirsen (SRP-4053) is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen can restore the expression of the anti-myostatin protein. Golodirsen can be used for the research of duchenne muscular dystrophy (DMD) .

HY-132611A

Golodirsen sodium SRP-4053 sodium		Antisense Oligonucleotides
Golodirsen (SRP-4053) sodium is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen sodium restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen sodium can restore the expression of the anti-myostatin protein. Golodirsen sodium can be used for the research of duchenne muscular dystrophy (DMD) .

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-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-177659

Rimigorsen		Antisense Oligonucleotides
Rimigorsen is an antisense oligonucleotide that induces skipping of exon 44 of the pre-mRNA encoding dystrophin in a Duchenne muscular dystrophy (DMD)

HY-177659A

Rimigorsen sodium		Antisense Oligonucleotides
Rimigorsen sodium is an antisense oligonucleotide that induces skipping of exon 44 of the pre-mRNA encoding dystrophin in a 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-127137A

Adenylosuccinic acid tetraammonium Adenylosuccinate tetraammonium; Aspartyl adenylate tetraammonium		Nucleotide 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-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 .

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Duchenne muscular dystrophy (DMD)

Inhibitors & Agonists

Inhibitory Antibodies

NaturalProducts

Oligonucleotides

Natural
Products