golodirsen

Golodirsen is an antisense oligonucleotide of phosphorodiamidate morpholino oligomer (PMO) chemistry, developed for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping. DMD is a severe X-linked muscle-wasting disorder caused by mutations in the dystrophin gene, leading to absence or dysfunction of the dystrophin protein, which is essential for muscle integrity. Golodirsen binds specifically to exon 53 of pre-mRNA from the DMD gene, causing this exon to be skipped during mRNA processing. This restores the reading frame and enables production of a truncated but partially functional dystrophin protein. The drug does not cure DMD but may slow disease progression and improve muscle function in about 8% of patients with relevant mutations. It is administered as a weekly intravenous infusion and was granted accelerated approval by the FDA in 2019 based on increased dystrophin levels rather than demonstrated clinical benefit[1][2][5][6][7][8].

Brand names

Vyondys 53Vyondys53Vyondys-53

Other names

golodirsen