Agents useful in the treatment of facioscapulohumeral muscular dystrophy

摘要

An antisense agent capable of binding to the pre-mRNA of the double homebox 4 (DUX4) or to the pre-mRNA of the double homebox 4c (DUX4c), in which the antisense agent is capable of binding to a sequence element necessary for the splicing of the pre-mRNA of DUX4 or DUX4c or is capable of binding to a sequence element necessary for the polyadenylation of the pre-mRNA of DUX4 or DUX4c, and in which the antisense agent is an antisense oligonucleotide or antisense oligonucleotide analog , for use as a medicine.