duchenne

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.

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.

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

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.

Edasalonexent (CAT-1004) is an orally available NF-κB inhibitor designed to improve Duchenne muscular dystrophy.

1-N-Methyl-4-mercaptohistidine disulfide is a potential Duchenne chloroplast coupling factor 1 redox regulator, an oxidized form of ochratoxin A, that inhibits light-triggered CF1 ATPase activity.

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.

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.

Adenylosuccinic acid (Adenylosuccinate) is a purine nucleotide cycle metabolite and a novel inducer of the Nrf2 cell protector. It can be used for research on Duchenne muscular dystrophy (DMD).

PMO-G is a spherical polymer micelle with skin permeability, capable of increasing dystrophin protein content in the abdominal muscle of mdx mice by up to 50-fold. PMO-G is utilized in research related to Duchenne muscular dystrophy.

Negamycin (hydrochloride) is a dipeptide antibiotic that can restore dystrophin expression in skeletal and cardiac muscles in the mdx mouse model of Duchenne muscular dystrophy (DMD). It binds to a segment of eukaryotic rRNA decoding site A. Negamycin (hydrochloride) is applicable in DMD research.

Naproxcinod is a derivative of naproxen exhibiting analgesic and anti-inflammatory activity. As a cyclooxygenase (COX)-inhibitory nitric oxide donor (CINOD), it is indicated for osteoarthritis and inflammatory conditions.

RTC13 is a read-through reagent that restores anti-myasthenia gravis protein expression in the mdx mouse model 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.

TG003 is a potent Clk1/Sty inhibitor that inhibits Clk1 and Clk4, inhibits cancer cell growth and induces apoptosis, and can be used in the study of Duchenne muscular dystrophy.

OX01914 is a water-soluble and permeable modulator of utrophin that upregulates utrophin protein levels with an EC50 of 20.5 μM. OX01914 can be used in Duchenne muscular dystrophy (DMD) studies [1].

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].

TG-693 is an orally active CLK1 (CDC2-like kinase 1) inhibitor that promotes exon 31 skipping of the dystrophin gene mutation in vivo and inhibits CLK1 substrate phosphorylation, suitable for studying Duchenne muscular dystrophy (DMD).

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].

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, improves cognitive performance in rats during the object location task (OLT), 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].

Suvodirsen sodium, which induces exon 51 skipping, has potential for studying Duchenne muscular dystrophy (DMD) [1].

TCL065, an ionizable cationic lipid (pKa = 6.3), is utilized in the creation of lipid nanoparticles (LNPs) for the delivery of mRNA and single-guide RNA (sgRNA) both in vitro and in vivo. When LNPs with TCL065 encapsulate sgRNA targeting the donor site of dystrophin exon 45, along with those targeting the splicing acceptor site of dystrophin exon 45, dystrophin expression is restored in myoblasts derived from induced-pluripotent stem (iPS) cells from Duchenne muscular dystrophy (DMD) patients. Additionally, TCL065-containing LNPs encapsulating mRNA encoding Cas9 nuclease, combined with those containing sgRNA targeting the Rosa26 locus, have been applied for CRISPR-mediated gene editing in mouse gastrocnemius muscle.