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Results for "

Duchenne

" in MedChemExpress (MCE) Product Catalog:

By Targets:	Androgen Receptor (1) Antibiotic (1) Antibody-Oligonucleotide Conjugates (AOCs) (1) Apoptosis (3) Arp2/3 Complex (1) Biochemical Assay Reagents (2) Calcium Channel (2) Cannabinoid Receptor (1) CDK (2) Cytochrome P450 (2) DNA/RNA Synthesis (7) Dystrophin (14) Endogenous Metabolite (4) Exosomes (1) Glucocorticoid Receptor (3) Guanylate Cyclase (1) HDAC (3) Liposome (2) Microtubule/Tubulin (2) Mineralocorticoid Receptor (2) Myosin (1) NF-κB (3) Nucleoside Antimetabolite/Analog (2) PPAR (1) Reference Standards (5) REV-ERB (1) TGF-beta/Smad (3) Transferrin Receptor (1) Transmembrane Glycoprotein (1)
By Research Areas:	Cancer (7) Cardiovascular Disease (4) Infection (1) Inflammation/Immunology (10) Metabolic Disease (18) Neurological Disease (13)
Oligonucleotides:	Nucleotide Analogs (1) Cationic Lipids (2) Antisense Oligonucleotides (18)

Inhibitors & Agonists

Biochemical Assay Reagents

Peptides

Inhibitory Antibodies

Natural
Products

Oligonucleotides

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

HY-14415

SR8278 Maximum Cited Publications 15 Publications Verification	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-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. Givinostat can penetrate the blood-brain barrier (BBB) .

HY-W015828

Ompenaclid 5+ Cited Publications 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-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-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-148799

Sevasemten EDG-5506	Myosin	Others
Sevasemten is an orally active, selective allosteric inhibitor of skeletal muscle myosin that protects skeletal muscle from contraction-induced injury. Sevasemten decreases muscle damage biomarkers and fibrosis while increasing muscle strength and activity in in Duchenne muscular dystrophy disease models .

HY-132611

Golodirsen SRP-4053	Dystrophin DNA/RNA Synthesis	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-P990051

Delpacibart AOC-1001 Antibody; AOC-1044 Antibody	Transferrin Receptor	Neurological Disease Inflammation/Immunology
Delpacibart is a humanized IgG1κ monoclonal antibody targeting the transferrin receptor TFRC. Delpacibart can be conjugated with the phosphorodiamidate morpholino oligonucleotide (PMO) Zotadirsen (HY-177972), which targets exon 44 of the dystrophin gene, to synthesize the antibody-oligonucleotide conjugate (AOC) Delpacibart zotadirsen (HY-177564). Delpacibart is suitable for use in Duchenne muscular dystrophy (DMD44) 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-119850

Aladorian ARM036; S44121	Calcium Channel	Cardiovascular Disease
Aladorian (ARM036; S44121) is a non-peptidic ryanodine receptor 2 (RyR2) stabilizer. Aladorian stabilizes RyR2 channels and rectifies abnormal Ca²⁺ handling in cardiomyocytes. Aladorian improves cardiomyocyte Ca²⁺ homeostasis independent of dystrophin restoration. Aladorian attenuates early cardiomyopathy and enhances left ventricular function in a canine muscular dystrophy model. Aladorian can be used for the research of heart failure, Duchenne muscular dystrophy-associated cardiomyopathy and muscular dystrophy .

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-P99370

Landogrozumab 1 Publications Verification 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 .

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-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-145724

Drisapersen Kyndrisa; GSK2402968A; PRO051	DNA/RNA Synthesis Dystrophin	Neurological Disease
Drisapersen (Kyndrisa) is a 2 ^'-O-methyl phosphorothioate RNA antisense oligonucleotide that induces exon 51 skipping. Drisapersen induces skipping of exon 51 during Dystrophin pre-mRNA splicing, allowing the synthesis of partially functional Dystrophin. Drisapersen can be used in research related to Duchenne muscular dystrophy .

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-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-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-18102

GLPG0492 5+ Cited Publications	Androgen Receptor	Neurological Disease
GLPG0492 is an orally active, non-steroidal selective androgen receptor modulator. GLPG0492 exerts functional transactivation by binding to the ligand-binding domain of the receptor, exhibiting preferential partial agonist activity in muscle and bone tissues with low activity in reproductive tissues. GLPG0492 effectively counteracts muscle atrophy-related pathways, significantly enhances muscle strength, maintains motor ability, reduces fibrosis and improves electrophysiological parameters. GLPG0492 prevents immobilization-induced muscle atrophy and regulates muscle mass homeostasis, serving as a valuable tool compound for studies on Duchenne muscular dystrophy, muscle loss and various types of disuse musculoskeletal atrophy .

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-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-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-147253

Brogidirsen NS 089; NCNP 02	DNA/RNA Synthesis	Neurological Disease
Brogidirsen (NS 089; NCNP 02) is a a dual-targeting antisense oligonucleotide. Brogidirsen can induce dystrophin protein experession. Brogidirsen can be used for the research of Duchenne muscular dystrophy .

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-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-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-14842R

Givinostat (Standard) ITF-2357 (Standard)	HDAC Reference Standards	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-153369

BAY-747 BAY 1165747	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 .

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-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-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-W015828R

Ompenaclid (Standard) RGX-202 (Standard); 3-Guanidinopropionic acid (Standard)	Reference Standards 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-176754

TCL065	Liposome	Metabolic Disease Inflammation/Immunology
TCL065 is an ionizable lipid with a pK_a of 6.3. TCL065 can be used to generate lipid nanoparticles (LNPs) for the delivery of mRNA as well as single-guide RNA (sgRNA) both in vitro and in vivo. TCL065-containing LNPs can studied in research for Duchenne muscular dystrophy and gene-editing technologies .

HY-109017R

Vamorolone (Standard) VBP15 (Standard)	Reference Standards Glucocorticoid Receptor Mineralocorticoid Receptor NF-κB	Inflammation/Immunology
Vamorolone (Standard) is the analytical standard of Vamorolone. This product is intended for research and analytical applications.

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-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-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-17614R

Ezutromid (Standard) SMT C1100 (Standard); BMN 195 (Standard); VOX-C1100 (Standard)	Cytochrome P450 Reference Standards	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-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-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-147253A

Brogidirsen sodium NS 089 sodium; NCNP 02 sodium	DNA/RNA Synthesis	Neurological Disease
Brogidirsen (NS 089; NCNP 02) sodium is a a dual-targeting antisense oligonucleotide. Brogidirsen sodium can induce dystrophin protein experession. Brogidirsen sodium can be used for the research of Duchenne muscular dystrophy .

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-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-W893751

PMO-G	Biochemical Assay Reagents	Metabolic Disease
PMO-G is a spherical-shape polymeric micelles. PMO-G has skin permeation. PMO-G can result in up to a 50-fold higher level of dystrophin in abdominal muscles of mdx mice. PMO-G can be studied in research on Duchenne muscular dystrophy .

HY-119850A

Aladorian sodium ARM036 sodium; S44121 sodium	Calcium Channel	Cardiovascular Disease
Aladorian (ARM036; S44121) sodium is a non-peptidic ryanodine receptor 2 (RyR2) stabilizer. Aladorian sodium stabilizes RyR2 channels and rectifies abnormal Ca²⁺ handling in cardiomyocytes. Aladorian sodium improves cardiomyocyte Ca ²⁺ homeostasis independent of dystrophin restoration. Aladorian sodium attenuates early cardiomyopathy and enhances left ventricular function in a canine muscular dystrophy model. Aladorian sodium can be used for the research of heart failure, Duchenne muscular dystrophy-associated cardiomyopathy and muscular dystrophy .

HY-101459R

RTC13 (Standard)	Dystrophin Reference Standards	Metabolic Disease
RTC13 (Standard) is the analytical standard of RTC13 (HY-101459). This product is intended for research and analytical applications. RTC13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy (DMD) .

HY-P11741

BV2	Exosomes Biochemical Assay Reagents	Neurological Disease
BV2 is a delivery peptide that binds to BVES, with a K_a of 2.03 μM for the BVES target. BV2 specifically binds to the extracellular domain of BVES, achieving muscle homing and cellular internalization via caveolae-mediated endocytosis. When BV2 is modified on the surface of exosomes by PMO, it enhances dystrophin restoration in the peripheral muscles and myocardium of dystrophin-deficient mice. BV2 is applicable to research related to Duchenne muscular dystrophy and muscle atrophy .

HY-185321

Tacadirsen	Dystrophin	Others
Tacadirsen is an oligonucleotide that can promote the synthesis of functional dystrophin synthesis, and is used for the research of Duchenne muscular dystrophy.

HY-185321A

Tacadirsen sodium	Dystrophin	Others
Tacadirsen sodium is an oligonucleotide that can promote the synthesis of functional dystrophin synthesis, and is used for the research of Duchenne muscular dystrophy.

Cat. No.	Product Name	Type

HY-147332

TCL053	Biochemical Assay Reagents
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-P11741

BV2	Exosomes Biochemical Assay Reagents	Neurological Disease
BV2 is a delivery peptide that binds to BVES, with a K_a of 2.03 μM for the BVES target. BV2 specifically binds to the extracellular domain of BVES, achieving muscle homing and cellular internalization via caveolae-mediated endocytosis. When BV2 is modified on the surface of exosomes by PMO, it enhances dystrophin restoration in the peripheral muscles and myocardium of dystrophin-deficient mice. BV2 is applicable to research related to Duchenne muscular dystrophy and muscle atrophy .

Cat. No.	Product Name	Target	Research Area	Image

HY-P990051

Delpacibart AOC-1001 Antibody; AOC-1044 Antibody	Transferrin Receptor	Neurological Disease Inflammation/Immunology
Delpacibart is a humanized IgG1κ monoclonal antibody targeting the transferrin receptor TFRC. Delpacibart can be conjugated with the phosphorodiamidate morpholino oligonucleotide (PMO) Zotadirsen (HY-177972), which targets exon 44 of the dystrophin gene, to synthesize the antibody-oligonucleotide conjugate (AOC) Delpacibart zotadirsen (HY-177564). Delpacibart is suitable for use in Duchenne muscular dystrophy (DMD44) research .

HY-P99370

Landogrozumab 1 Publications Verification 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-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-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-P992223

Anti-DAG1/Dystroglycan Antibody (DAG-6F4)	Transmembrane Glycoprotein	Neurological Disease
Anti-DAG1/Dystroglycan Antibody (DAG-6F4) is a human monoclonal antibody against DAG1/Dystroglycan. Anti-DAG1/Dystroglycan Antibody (DAG-6F4) can bind specifically to the α and β subunits of the dystroglycan protein complex. Anti-DAG1/Dystroglycan Antibody (DAG-6F4) is applicable to research related to muscular dystrophy .

Cat. No.	Product Name		Classification

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-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-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-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 .

HY-145724

Drisapersen Kyndrisa; GSK2402968A; PRO051		Antisense Oligonucleotides
Drisapersen (Kyndrisa) is a 2 ^'-O-methyl phosphorothioate RNA antisense oligonucleotide that induces exon 51 skipping. Drisapersen induces skipping of exon 51 during Dystrophin pre-mRNA splicing, allowing the synthesis of partially functional Dystrophin. Drisapersen can be used in research related to Duchenne muscular dystrophy .

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-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-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-147253

Brogidirsen NS 089; NCNP 02		Antisense Oligonucleotides
Brogidirsen (NS 089; NCNP 02) is a a dual-targeting antisense oligonucleotide. Brogidirsen can induce dystrophin protein experession. Brogidirsen can be used for the research of Duchenne muscular dystrophy .

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-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-132585A

Vesleteplirsen sodium SRP-5051 sodium		Antisense Oligonucleotides
Vesleteplirsen (SRP-5051) sodium is a next-generation antisense oligonucleotide of peptide phosphorodiamidate morpholino oligomer (PPMO). Vesleteplirsen targets exon 51 skipping in 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-176754

TCL065		Cationic Lipids
TCL065 is an ionizable lipid with a pK_a of 6.3. TCL065 can be used to generate lipid nanoparticles (LNPs) for the delivery of mRNA as well as single-guide RNA (sgRNA) both in vitro and in vivo. TCL065-containing LNPs can studied in research for Duchenne muscular dystrophy and gene-editing technologies .

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-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-147253A

Brogidirsen sodium NS 089 sodium; NCNP 02 sodium		Antisense Oligonucleotides
Brogidirsen (NS 089; NCNP 02) sodium is a a dual-targeting antisense oligonucleotide. Brogidirsen sodium can induce dystrophin protein experession. Brogidirsen sodium can be used for the research of Duchenne muscular dystrophy .

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-185321

Tacadirsen		Antisense Oligonucleotides
Tacadirsen is an oligonucleotide that can promote the synthesis of functional dystrophin synthesis, and is used for the research of Duchenne muscular dystrophy.

HY-185321A

Tacadirsen sodium		Antisense Oligonucleotides
Tacadirsen sodium is an oligonucleotide that can promote the synthesis of functional dystrophin synthesis, and is used for the research of Duchenne muscular dystrophy.

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