Neuromuscular Disease

Baliforsen (ISIS 5987690) is an antisense oligonucleotide (ASO) that inhibits DMPK mRNA. Baliforsen binds within exon 9 of the human DMPK transcript to promote RNase H1-mediated degradation Baliforsen can be used for the research of myotonic dystrophy type 1.

DC-98-LC74 is a selective modulator of adult skeletal muscle-type nicotinic acetylcholine receptor (α1β1δε), with an EC₅₀ value of 6.5 µM for the human receptor and an IC₅₀ of 13.45 µM for the human α3β4 nicotinic acetylcholine receptor. DC-98-LC74 increases the ligand-free opening probability of the receptor via the ε subunit M2-M3 loop, and prolongs the burst duration and opening probability of wild-type and fast-channel mutant AChR. DC-98-LC74 exerts weak effects on neuronal AChR subtypes and has no agonist activity. DC-98-LC74 prolongs the mouse diaphragm endplate current and improves muscle contractility in isolated neuromuscular preparations from sarcopenic mice. DC-98-LC74 can be used for studies on neuromuscular junction function and myasthenia-related diseases.

Amogammadex (Adamgammadex; Aom0498) is a modified γ-cyclodextrin (HY-W040040) derivative and neuromuscular blocking agent. Amogammadex forms inactive, tight inclusion complexes with free Rocuronium (HY-17033) and Vecuronium (HY-B0118A), and reverses the neuromuscular blockade induced by them in a concentration-dependent manner. Amogammadex can be used in research related to neuromuscular blockade reversal.