Sarepta Therapeutics, a leader in precision genetic medicine for rare diseases, has recently achieved a significant milestone with the FDA's approval of AMONDYS 45 (casimersen). This antisense oligonucleotide, developed from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, is now approved for treating Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 45 skipping. This approval follows a demonstration of a statistically significant increase in dystrophin production in skeletal muscle, which is expected to provide clinical benefits for exon 45-amenable patients. Continued approval of AMONDYS 45 may depend on the res...
