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Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!2025-02-06
151 Results for "
dystrophy
" in MedChemExpress (MCE) Product Catalog:
Products (151)
151 Results for "dystrophy" in MCE Product Catalog:
Cat. No.: HY-P99857
CAS No.: 1629605-31-7
Synonyms: PF-06252616
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Cat. No.: HY-17614
CAS No.: 945531-77-1
Synonyms: SMT C1100; BMN 195; VOX-C1100
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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 .
Cat. No.: HY-P990993
CAS No.: 2922218-19-5
Synonyms: GYM-329, RG6237, RG-70240
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Synonyms: Kyndrisa; GSK2402968A; PRO051
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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 .
Cat. No.: HY-145725A
CAS No.: 1698048-23-5
Synonyms: ISIS 598769; IONIS 598769; BIIB 065; ISIS-DMPK-2.5Rx
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Baliforsen (ISIS 5987690) is an antisense oligonucleotide (ASO) that inhibits DMPK mRNA. Baliforsen binds within exon 9 of the human DMPK transcript to promote RNase H1-mediated degradation Baliforsen can be used for the research of myotonic dystrophy type 1 .
Cat. No.: HY-132611A
Purity: 92.00%
Synonyms: SRP-4053 sodium
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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) .
Cat. No.: HY-108753A
Purity: 91.16%
Synonyms: AVI 4658 sodium
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Eteplirsen (AVI 4658) sodium is a phosphorylated diamine morpholino oligonucleotide that targets exon 51 of the human Duchenne muscular dystrophy (DMD) gene. Eteplirsen sodium induces exon 51 skipping, causing it to be skipped during splicing, thereby restoring the translation reading frame and producing a shortened functional dystrophin. Eteplirsen sodium can be used in research on Duchenne muscular dystrophy .
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 .
Synonyms: ASP0367; MA-0211
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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) .
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DNA, d(P-thio)([2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rC-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]m5C-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]m5C-G-A-A-T-G-T-m5C-m5C-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]G-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]A-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA), sodium salt (1:15)
Synonyms: IONIS 598769 sodium; ISIS 598769 sodium
Cat. No.: HY-16361A
CAS No.: 200189-97-5
Synonyms: CGP3466B; CGP3446 maleate; TCH346 maleate
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Neurological Disease Metabolic Disease Inflammation/Immunology
Omigapil maleate (CGP3466B), an orally active GAPDH nitrosylation inhibitor, abrogates Aβ1-42-induced tau acetylation, memory impairment, and locomotor dysfunction in mice. Omigapil maleate has the potential for the research of Alzheimer's disease. Omigapil maleate is a apoptosis inhibitor. Omigapil maleate can be used for the research of congenital muscular dystrophy (CMD). Omigapil maleate is a click chemistry reagent, it contains an Alkyne group and can undergo copper-catalyzed azide-alkyne cycloaddition (CuAAc) with molecules containing Azide groups .
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Cat. No.: HY-P10242
CAS No.: 1621169-56-9
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Cat. No.: HY-132586A
Purity: 95.25%
Synonyms: NS-065/NCNP-01 sodium
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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 .
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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 .
Cat. No.: HY-147253
CAS No.: 2409088-11-3
Synonyms: NS 089; NCNP 02
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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 .
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RTC13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy (DMD) .
Cat. No.: HY-122970
CAS No.: 77769-21-2
Synonyms: 1,2-Dihydrotanshinquinone
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Labiatae Terpenoids Samanea saman (Jacq.) Merr. Diterpenoids Plants Disease Research Fields Inflammation/Immunology
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Cat. No.: HY-127137A
Purity: ≥98.0%
Synonyms: Adenylosuccinate tetraammonium; Aspartyl adenylate tetraammonium
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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) .
Cat. No.: HY-161050
CAS No.: 3032969-58-4
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 .
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Protocols
Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!A suitable model is crucial in drug screening experiments. Organs can mimic the three-dimensional functional structure of internal organs, have similar spatial organization to corresponding organs, maintain some key characteristics, and reproduce some physiological functions. They are widely used for modeling and personalized drug screening physiological functions.2025-02-06
