Tofersen (BIIB067) is an antisense oligonucleotide that mediates RNase H-dependent degradation of superoxide dismutase 1 (SOD1) mRNA to reduce SOD1 protein synthesis. Tofersen can be used in amyotrophic lateral sclerosis (ALS) research.
|Solubility (25°C)||Water ≥ 60 mg/mL|
|Storage||-20°C, dry, sealed|
|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.
 Timothy M Miller, et al. N Engl J Med. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
 Johannes Dorst, et al. Curr Opin Neurol. Clinical studies in amyotrophic lateral sclerosis
 Michael Benatar, et al. Neurotherapeutics. Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study
 Daisuke Ito. Ann Neurol. Promise of Nucleic Acid Therapeutics for Amyotrophic Lateral Sclerosis
 Timothy Miller, et al. N Engl J Med. Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
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