The exon-skipping drug was approved by the FDA for DMD patients with mutations in the dystrophin gene amenable to exon 53 skipping, a group that accounts for around 8% of the total DMD population.

Recent trends in DMD research include a strong focus on advanced therapeutic strategies, such as gene editing, exon-skipping therapies, and systemic delivery methods for mRNA. According to the ...

The therapy is designed to skip exon 51 of the dystrophin gene, which is mutated in approximately 13% of patients with DMD. Earlier this year, PepGen shared results from the first dose cohort (5 ...

Of note, exon-skipping oligonucleotides and gene therapy in DMD have been approved under the FDA's accelerated approval program, using a surrogate marker instead of clinical benefit as an endpoint.

Sarepta's exon-skipping therapies for DMD – Exondys 51, Vyondys 53 (golodirsen), and Amondys 45 (casimersen) – have been the main drivers of Sarepta's revenue growth in recent years ...

The CONNECT2-EDO51 trial is designed to test PepGen’s PGN-EDO51 exon-skipping therapy over 25 weeks in patients with DMD. In a short press release Monday morning, the biotech didn’t offer any ...