myotonic

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.

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.

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.

(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.

Pip6a is an arginine-rich cell-penetrating peptide that facilitates the transport of drugs across plasma and endosomal membranes. It consists of a hydrophobic core flanked by arginine-rich domains containing β-alanine and aminocaproic acid spacers. When Pip6a is combined with morpholino phosphorodiamidate oligomer (PMO), it significantly enhances the delivery of antisense oligonucleotides (ASO) to the striated muscle of myotonic dystrophy type 1 (DM1) mice.