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).

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.

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.

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.

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.

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 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).

RNA binder 2 (Compound 20) serves as a covalent inhibitor of r(CUG)expRNA in myotonic dystrophy type 1 (DM1). It attaches to RNA through the Hoechst scaffold, hindering the binding of MBNL1 and reinstating the normal splicing process. This compound holds potential for research in the context of myotonic dystrophy type 1.

Deglycobleomycin is an RNA cleaving agent that targets r(CUG) repeat sequences. By binding with small molecules that recognize r(CUG)exp, Deglycobleomycin can selectively cleave r(CUG)exp. Its efficiency in cleaving DNA is considerably lower than that of Bleomycin A5(Pingyangmycin), but it exhibits enhanced selectivity for r(CUG)exp. Deglycobleomycin is useful for research on diseases associated with r(CUG) repeat expansions, such as myotonic dystrophy type 1, and the development of drugs aimed at precise targeting and cleavage of pathogenic RNA.

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.

Basivarsen linker is a connector employed in Zeleciment basivarsen, facilitating the attachment of a Fab antibody (Zeleciment) that targets the TfR1 receptor to an antisense oligonucleotide. Zeleciment basivarsen is studied for its potential application in the treatment of myotonic dystrophy type 1 (DM1).

Zeleciment basivarsen (DYNE-101) is an antibody oligonucleotide conjugate (AOC), consisting of a Fab antibody named Zeleciment that binds to the transferrin receptor 1 (TfR1) on muscle cells, and an antisense oligonucleotide known as Basivarsen. This compound targets the RNA of the mutant dystrophia myotonica-protein kinase (DMPK) for RNase H-mediated degradation, and is utilized in the study of myotonic dystrophy type 1 (DM1).

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 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.

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.