Scientific Reviews
Product Guides
Blogs
Topics
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
36 Results for "
Duchenne muscular dystrophy (DMD)
" in MedChemExpress (MCE) Product Catalog:
Products (36)
36 Results for "Duchenne muscular dystrophy (DMD)" in MCE Product Catalog:
12
Target:
Research Areas:
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) .
Synonyms: AVI 4658
Target:
Research Areas:
Eteplirsen (AVI 4658) is a phosphorylated diamine morpholino oligonucleotide that targets exon 51 of the human Duchenne muscular dystrophy (DMD) gene. Eteplirsen induces exon 51 skipping, causing it to be skipped during splicing, thereby restoring the translation reading frame and producing a shortened functional dystrophin. Eteplirsen can be used in research on Duchenne muscular dystrophy .
Synonyms: SRP-4053
Target:
Research Areas:
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) .
Cat. No.: HY-P990051
CAS No.: 2653183-66-3
Synonyms: AOC-1001 Antibody; AOC-1044 Antibody
Research Areas:
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 .
Cat. No.: HY-132586
CAS No.: 2055732-84-6
Synonyms: NS-065/NCNP-01
Research Areas:
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 .
Synonyms: Adenylosuccinate; Aspartyl adenylate
Category:
Human Gut Microbiota Metabolites Microorganisms Metabolic Disease Endogenous metabolite Disease Research Fields
Target:
Cat. No.: HY-P99857
CAS No.: 1629605-31-7
Synonyms: PF-06252616
Target:
Research Areas:
Cat. No.: HY-17614
CAS No.: 945531-77-1
Synonyms: SMT C1100; BMN 195; VOX-C1100
Target:
Research Areas:
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-132611A
Purity: 92.00%
Synonyms: SRP-4053 sodium
Target:
Research Areas:
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
Target:
Research Areas:
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 .
Synonyms: ASP0367; MA-0211
Target:
Research Areas:
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) .
Cat. No.: HY-132586A
Purity: 95.25%
Synonyms: NS-065/NCNP-01 sodium
Research Areas:
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 .
Target:
Research Areas:
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 .
Target:
Research Areas:
RTC13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy (DMD) .
0 Images
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-127137A
Purity: ≥98.0%
Synonyms: Adenylosuccinate tetraammonium; Aspartyl adenylate tetraammonium
Target:
Research Areas:
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-14842R
CAS No.: 497833-27-9
Synonyms: ITF-2357 (Standard)
Target:
Research Areas:
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 .
Cat. No.: HY-132584A
Purity: 99.07%
Synonyms: SRP-4045 sodium
Target:
Research Areas:
Casimersen (SRP-4045) 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) .
Cat. No.: HY-132585A
Synonyms: SRP-5051 sodium
Research Areas:
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 .
Cat. No.: HY-153369
CAS No.: 1609342-18-8
Synonyms: BAY 1165747
Target:
Research Areas:
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 .
- 1
- 2
-
-
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
