FIELD: biotechnology.
SUBSTANCE: described is a group of inventions comprising a compound which promotes production in a target cell of a mutant activin receptor type 2B (ACVR2B), or a pharmaceutically acceptable salt or hydrate thereof (embodiments), a pharmaceutical composition for use in the prevention or treatment of amyotrophic disease, muscular dystrophy or sarcopenia, method of treating amyotrophic disease, muscular dystrophy or sarcopenia and use of a compound or a pharmaceutically acceptable salt thereof or a hydrate for the purpose of preparing a drug for preventing or treating amyotrophic disease, muscular dystrophy or sarcopenia.
EFFECT: invention extends the range of agents promoting production of mutant activin receptor type 2B (ACVR2B) in the target cell.
29 cl, 3 dwg, 8 tbl, 5 ex
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