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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-16918A
CAS No.: 161374-55-6
Synonyms: SBi4211 dimethanesulfonate
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Heptamidine dimethanesulfonate (SBi4211 dimethanesulfonate) is a potent Pentamidine-related inhibitor of the calcium-binding protein S100B (Kd=6.9 μM), selectively kills melanoma cells with S100B over those without S100B . Heptamidine is a useful tool for the investigation of Myotonic dystrophy (DM) .
Cat. No.: HY-177454
CAS No.: 2763758-01-4
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Basivarsen linker is a linker used in HY-177452 Zeleciment basivarsen for coupling a TfR1-binding Fab Zeleciment (HY-P990780) and an antisense oligonucleotide. Zeleciment basivarsen is an antibody-oligonucleotide conjugate (AOC) designed to target mutant nuclear myotonic dystrophy protein kinase (DMPK) RNA for RHase H-mediated degradation to correct splicing. It is used for the study of myotonic dystrophy type 1 (DM1).
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RNA, (P-thio)(Um-Cm-Am-Am-Gm-Gm-Am-Am-Gm-Am-Um-Gm-Gm-Cm-Am-Um-Um-Um-Cm-Um), sodium salt
Synonyms: Kyndrisa sodium; GSK2402968A sodium; PRO051 sodium
Cat. No.: HY-111423
CAS No.: 2226507-05-5
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BDP8900 is a potent and selective inhibitor of myotonic dystrophy-related Cdc42-binding kinases (MRCKα and MRCKβ). BDP8900 reduces substrate phosphorylation, leading to morphological changes, motility inhibition and invasiveness of cancer cells .
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MA-0204 is a potent, highly selective and orally available peroxisome proliferator activated receptor δ (PPARδ) modulator with EC50s of 0.4 nM, 7.9 nM and 10 nM for human, mouse and rat PPARδ, respectively. Potential treatment for Duchene Muscular Dystrophy (DMD) .
Cat. No.: HY-132585A
Synonyms: SRP-5051 sodium
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Vesleteplirsen sodium (SRP-5051 sodium) is a peptide-conjugated morpholino oligonucleotide (PPMO) that mediates exon jumping molecular effects. Vesleteplirsen sodium targets exon 51 of the DMD gene pre-mRNA, remodeling the transcript splicing process, restoring the protein translation read frame, and generating internally truncated pseudodystrophy proteins. Vesleteplirsen sodium can be used for research related to Duchenne muscular dystrophy .
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(R)-BDP9066 is an isomer of BDP9066 and has lower activity compared to BDP9066. BDP9066 is a potent inhibitor of myotonic dystrophy kinase-related Cdc42-binding kinase (MRCK). (R)-BDP9066 is derived from patent WO2019034890A1 (E118) .
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Activated DPG Subunit can be used in the synthesis of exon jumping oligomer conjugates. The oligomer conjugates complement selected target sites in the human anti-muscular atrophy protein gene and induce exon 51 jumping. It can be used for research of muscular dystrophy .
Cat. No.: HY-14842R
CAS No.: 497833-27-9
Synonyms: ITF-2357 (Standard)
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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 .
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Activated T Subunit can be used in the synthesis of exon jumping oligomer conjugates. The oligomer conjugates complement selected target sites in the human anti-muscular atrophy protein gene and induce exon 51 jumping. Activated T subunit can be used for research of muscular dystrophy .
Cat. No.: HY-114898
CAS No.: 1826026-00-9
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ML404 (Compound 38) is an inhibitor of the mitochondrial permeability transition pore (mtPTP), which can suppress mitochondrial swelling (EC50=4.9 nM) and only disrupts mitochondrial coupling (an adverse effect) at concentrations > 100 μM. ML404 can be utilized in research related to multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), muscular dystrophies (MD), myocardial infarction, and stroke .
Cat. No.: HY-P10567
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Cat. No.: HY-177971
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Braxlosiran is a component of the AOC reagent Delpacibart braxlosiran (HY-177566), consisting of an siRNA targeting DUX4 (double homeobox protein) and SMCC linker (HY-42360). Delpacibart braxlosiran binds to TfR1. Aberrant expression of the DUX4 protein causes changes in gene expression in muscle cells, which are associated with muscle function loss in facioscapulohumeral muscular dystrophy (FSHD) .
Cat. No.: HY-177659
CAS No.: 1196915-71-5
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Rimigorsen is an antisense oligonucleotide that induces skipping of exon 44 of the pre-mRNA encoding dystrophin in a Duchenne muscular dystrophy (DMD)
Cat. No.: HY-150077
CAS No.: 3068935-32-7
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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) .
Cat. No.: HY-48959
CAS No.: 849121-82-0
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α7β1 integrin modulator-1 is a potent α7β1 integrin modulator. α7β1 integrin modulator-1 has the potential for the research of muscular dystrophy .
Cat. No.: HY-122588A
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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 .
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RNA, ((2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)C-[P(S)]-sp-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-(2'-deoxy-2'-fluoro)G-[P(S)]-sp-(2'-deoxy-2'-fluoro)G-[P(S)]-sp-Am-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-Gm-Am-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-Gm-Gm-(2'-deoxy-2'-fluoro)C-[P(S)]-sp-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)C-[P(S)]-sp-(2'-deoxy-2'-fluoro)U), sodium salt
Cat. No.: HY-132592A
CAS No.: 2142024-01-7
Synonyms: WVE-210201 sodium
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RNA, ((2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)C-[P(S)]-sp-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-(2'-deoxy-2'-fluoro)G-[P(S)]-sp-(2'-deoxy-2'-fluoro)G-[P(S)]-sp-Am-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-Gm-Am-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-Gm-Gm-(2'-deoxy-2'-fluoro)C-[P(S)]-sp-(2'-deoxy-2'-fluoro)A-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)U-[P(S)]-sp-(2'-deoxy-2'-fluoro)C-[P(S)]-sp-(2'-deoxy-2'-fluoro)U)
Cat. No.: HY-132592
CAS No.: 2095270-67-8
Synonyms: WVE-210201
Cat. No.: HY-17614R
CAS No.: 945531-77-1
Synonyms: SMT C1100 (Standard); BMN 195 (Standard); VOX-C1100 (Standard)
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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 .
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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
