duchenne muscular dystrophy

ART-CHEM-BB B025267 is the upregulator of utrophin production with EC50 of 1.8 μM and can be used in research on the treatment of Duchenne muscular dystrophy.

Edasalonexent (CAT-1004) is an orally available NF-κB inhibitor for the improvement of Duchenne muscular dystrophy.

SR8278 is an REV-ERBα antagonist(EC50 = 0.47 µM), blocking activation of the receptor by the synthetic agonist GSK 4112

Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide utilized in research for Duchenne muscular dystrophy.

Adenylosuccinic acid (Adenylosuccinate; Aspartyl adenylate) is a purine ribonucleoside monophosphate and plays a role in nucleotide cycle metabolite. Adenylosuccinic acid has the potential for the study of duchenne muscular dystrophy(DMD)[1] and can be co

Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide that specifically targets the splicing of exon 53 in the dystrophin gene. It is utilized in research related to Duchenne muscular dystrophy (DMD).

Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind and target exon 53 of dystrophin pre-mRNA, with applications in the investigation of Duchenne muscular dystrophy (DMD).

BAY-747 (BAY 1165747) is an orally administered, brain-penetrant compound that stimulates soluble guanylate cyclase (sGC). It effectively counteracts memory impairments induced by L-NAME and improves cognitive performance in rats during the object location task (OLT). Additionally, BAY-747 reduces blood pressure in conscious normotensive and spontaneously hypertensive rats (SHR) and enhances skeletal muscle function in the mdx/mTRG2 mouse model of Duchenne muscular dystrophy (DMD) [1] [2] [3].

RTC13, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy.

Naproxcinod, a nitric oxide-releasing NSAID derivative, significantly improved skeletal muscle strength and fatigue resistance in sedentary and exercise mice, reducing inflammatory infiltration and fibrotic deposition in the myocardium and diaphragm. In a

Vamorolone (VBP15) is an orally active dissociative steroidal anti-inflammatory drug and membrane-stabilizer. Vamorolone improves muscular dystrophy without side effects. Vamorolone displays effective NF-κB inhibition and substantially decreases hormonal effects.

OX01914 is a water-solube and permeable modulator of utrophin that can upregulate utrophin protein levels with an EC 50 of 20.5 μM. OX01914 can be used in Duchenne muscular dystrophy (DMD) study [1].

Drisapersen, an antisense oligonucleotide, facilitates exon 51 skipping in dystrophin pre-mRNA splicing, enabling the production of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with suitable mutations.

Suvodirsen (WVE-210201) is an oligonucleotide compound with potential applications in the study of Duchenne muscular dystrophy (DMD).

RTC14, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy.

Ataluren (PTC124) is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk.

Bocidelpar is a modulator of PPARδ with an EC50 of 7.80 nM and improves mitochondrial biogenesis and function in Duchenne Muscular Dystrophy muscle cells.

SR 12460 is a mimetic of the IκB kinase β (IKKβ) NEMO/IKKγ-binding domain (NBD) that inhibits the protein-protein interaction between the IKK complex subunits NF-κB essential modulator (NEMO/IKKγ) and IKKβ.1It inhibits TNF-α-induced NF-κB activation in a reporter assay when used at concentrations ranging from 25 to 150 μM. It reduces LPS-induced production of IL-6 in RAW 264.7 cells. SR 12460 (30 mg/kg) improves grip strength in themdxmouse model of Duchenne muscular dystrophy. 1.Zhao, J., Zhang, L., Mu, X., et al.Development of novel NEMO-binding domain mimetics for inhibiting IKK/NF-kB activationPLoS Biol.16(6)e2004663(2018)

TG693, an orally available inhibitor of CLK1, promotes the skipping of the endogenous mutated exon 31 in Duchenne muscular dystrophy (DMD) patient-derived cells and increased the production of the functional exon 31-skipped dystrophin protein.

Utrophin Modulator 1 (UM1) is a highly effective agent that upregulates utrophin protein levels, displaying an EC 50 of 0.11 μM. Its application in the investigation of Duchenne Muscular Dystrophy (DMD) has been well-established [1].

Casimersen (SRP-4045) is an antisense oligonucleotide, specifically belonging to the phosphorodiamidate morpholino oligomer subclass. It binds to exon 45 of dystrophin pre-mRNA, effectively restoring the open-reading frame by skipping exon 45. This action ultimately leads to the production of a functional dystrophin protein, albeit internally truncated. Casimersen finds utility in Duchenne muscular dystrophy (DMD) research.

Adenylosuccinic Acid Tetraammonium (also known as Adenylosuccinate or Aspartyl Adenylate) is an orally active compound that functions as a purine ribonucleoside monophosphate and is involved in nucleotide cycle metabolism. It can be transformed into fumaric acid via adenylosuccinate lyase action. This compound holds potential for research in Duchenne Muscular Dystrophy (DMD) [1].

Ezutromid (BMN 195) is a translation modulator of Small utrophin (EC50: 0.4 uM). Ezutromid is an orally bioavailable small molecule that transcriptionally upregulates the utrophin gene, increasing both utrophin mRNA and protein. Through this action, Ezutromid increases muscle function in dystrophin-deficient MDX mice, a mouse model of muscular dystrophy.