Duchenne muscular dystrophy

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

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

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).

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.

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.

Zotadirsen is a phosphorodiamidate morpholino oligomer (PMO) that targets exon 44 of the dystrophin gene. It is utilized in the synthesis of AOC drugs, such as Delpacibart zotadirsen, and is applicable in the study of Duchenne muscular dystrophy (DM).

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.

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.

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.

Eteplirsen (brand name Exondys 51) is an antisense oligonucleotide therapy designed to induce exon 51 skipping in the dystrophin gene, enabling partial restoration of dystrophin protein expression in patients with Duchenne muscular dystrophy carrying specific exon 51 mutations, Eteplirsen is extensively applied in genetic disease research, RNA-targeted therapy development, and translational studies of muscular dystrophy.

Casimersen (SRP-4045) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer class that binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame through exon 45 skipping. Casimersen enables the production of an internally truncated yet functional dystrophin protein, thereby serving as a specialized molecular tool for studying exon-skipping mechanisms and therapeutic development in Duchenne muscular dystrophy research.

Golodirsen (SRP-4053) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) class that restores the dystrophin gene reading frame in Duchenne muscular dystrophy by modulating pre-mRNA splicing to induce skipping of exon 53, thereby enabling partial dystrophin expression and restoring anti-myostatin protein levels, which supports its application as a molecular tool for studying exon-skipping strategies and disease-modifying approaches in DMD research.

Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide that specifically binds exon 53 of the dystrophin mRNA precursor, restoring the amino acid open-reading frame through exon 53 skipping and resulting in the production of a shortened but functionally relevant dystrophin protein. Viltolarsen provides a mechanistically precise experimental agent for investigating exon-skipping therapies and molecular pathology in Duchenne muscular dystrophy research.

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].