PG 01 is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. Corrects gating defects of CFTR mutants such as ΔF508 (Ka = 0.3 μM), E193K and G970R (Kd values are 0.22 μM and 0.45 μM respectively). Increases ΔF508-CFTR Cl- currents in the presence of forskolin; displays no effect on Ca2+-activated Cl- current.
|Solubility (25°C)||DMSO 43.95 mg/mL|
Powder -20°C 3 years ; 4°C 2 years
In solvent -80°C 6 months ; -20°C 1 month
|Body Surface Area (m2)||0.007||0.025||0.15||0.05||0.02||0.5|
|Animal A (mg/kg) = Animal B (mg/kg) multiplied by||Animal B Km|
|Animal A Km|
For example, to modify the dose of Compound A used for a mouse (20 mg/kg) to a dose based on the BSA for a rat, multiply 20 mg/kg by the Km factor for a mouse and then divide by the Km factor for a rat. This calculation results in a rat equivalent dose for Compound A of 10 mg/kg.
Olezarsen, an N-acetylgalactosamine (GalNAc)-suffixed antisense oligonucleotide (ASO) targeting hepatic APOC3 mRNA, inhibits the production of apolipoprotein C-III (apoC-III) and can be used in studies related to familial celiac sprue syndrome (FCS).
Lumasiran is a siRNA compound that reduces hepatic oxalate production by targeting ethanoic acid oxidase.
Givosiran is a small interfering RNA that targets hepatic aminofibrillar synthase 1 (ALAS1) messenger RNA, down-regulates ALAS1 mRNA, and prevents the accumulation of neurotoxic Î±-aminofibrillar acids and porphyrins, which can be used in porphyria-related studies.
Viltolarsen is a phosphorodiamidomorpholine antisense oligonucleotide that targets splicing of exon 53 of the myotonic dystrophy protein gene and can be used in the study of Duchenne muscular dystrophy (DMD).
Golodirsen is a phosphodiesteric morpholino oligomer (PMO) that specifically targets exon 53 of the pre-protein mRNA of myotonic dystrophy protein culture proteins, and can be used in studies related to Duchenne muscular dystrophy (DMD).
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