Casimersen

Casimersen, sold under the brand name Amondys 45, is an antisense oligonucleotide medication used for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping It is an antisense oligonucleotide of phosphorodiamidate morpholino oligomer (PMO).Duchenne muscular dystrophy is a rare disease that primarily affects boys.[5] It is caused by low levels of a muscle protein called dystrophin.[5] The lack of dystrophin causes progressive muscle weakness and premature death.