dystrophy

Neuromuscular-targeting compound 1 can be used to study muscular dystrophy and neuromuscular-related diseases.

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

CGP 3466B maleate (Omigapil maleate) is an orally bioavailable GAPDH nitrosylation inhibitor. Omigapil maleate abrogates Aβ1-42-induced tau acetylation, memory impairment, and locomotor dysfunction in mice. Omigapil maleate has the potential for the research of Alzheimer's disease. Omigapil maleate (CGP3446B maleate) is a apoptosis inhibitor. Omigapil maleate can be used for the research of congenital muscular dystrophy (CMD).

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.

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

LM11A-31 dihydrochloride is a water-soluble, non-peptide with high blood-brain barrier permeability.LM11A-31 dihydrochloride, a p75NTR (neurotrophin receptor p75) Ligand, is a potent proNGF (nerve growth factor) antagonist.

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.

Heptamidine dimethanesulfonate (SBi4211 dimethanesulfonate) serves as a potent inhibitor related to Pentamidine, targeting the calcium-binding protein S100B with a dissociation constant (Kd) of 6.9 µM. It demonstrates specificity by preferentially killing melanoma cells overexpressing S100B compared to cells lacking this protein. Additionally, Heptamidine is employed as a valuable research tool in the study of Myotonic dystrophy (DM).

Heptamidine is an effective Pentamidine-related inhibitor of the calcium-binding protein S100B (Kd: 6.9 μM). It selectively kills melanoma cells with S100B over those without S100B. It is a useful tool for the investigation of Myotonic 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).

MA-0204 is a highly selective and orally available peroxisome proliferator-activated receptor δ (PPARδ) modulator (EC50s: 0.4 nM, 7.9 nM and 10 nM for human, mouse and rat PPARδ, respectively). It is a potential treatment for Duchene 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.

Mexiletine is a non-selective voltage-gated sodium channel blocker. It is a medicine in the class of IB anti-arrhythmic. Mexiletine may also be used in patients experiencing refractory pain and is effective to treat muscle stiffness resulting from myotonic dystrophy or non-dystrophic myotonias.

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

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.

6-Aminophenanthridine is an antiprion agent. It inhibits prion formation in yeast- and mammalian-based screening assays when used alone and, to a greater extent, when used in combination with the α2-adrenergic receptor agonist guanabenz . 6-Aminophenanthridine (300 μM) inhibits protein folding activity of the ribosome (PFAR) by directly competing with protein substrates for the active site and decreases the yield of refolded protein without affecting the refolding rate. It prevents progressive wing position defects in a Drosophila model of oculopharyngeal muscular dystrophy (OPMD) when larvae are raised on medium containing doses ranging from 300 to 400 μM and in adults following dietary administration of 1-3 mM doses. 6-Aminophenanthridine also reduces muscle degeneration and decreases the number of nuclear inclusions in thoracic muscle in a Drosophila model of OPMD.

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.

Poloxamer 188 is a nonionic triblock copolymer surfactant and bioactive excipient used as a medical excipient to exacerbate cerebral amyloidosis, presynaptic dystrophy, and pathogenic microglial cell activation in 5XFAD mice.

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].

(R)-BDP9066 is a highly effective inhibitor of myotonic dystrophy kinase-related Cdc42-binding kinase (MRCK), effectively impeding cancer cell invasion, making it an invaluable resource for studying and researching proliferative diseases, particularly cancer.

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].