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.

“With current exon-skipping techniques, sometimes not all of the exon gets skipped, so there’s still part of the sequence we ...

The drug – formerly known as SRP-5051 – is a more potent version of Exondys 51 (eteplirsen), an exon-skipping therapy that can be used to treat around 13% of the population with the muscle ...

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

Results from the Forward-53 trial (NCT04906460) show the exon-skipping oligonucleotide-based therapy improved muscle health by the end of week 48 including a decline in necrosis and inflammation.

Sustained exon skipping, dystrophin restoration, and improved muscle indicators were observed, including reduced inflammation and fibrosis. A 3.8-second improvement in time to rise (TTR ...

None of the eteplirsen-treated patients reached a left ventricular ejection fraction below 50% compared with 22.1% of patients in the control group.

The latest 48-week data come from the Forward-53 trial of Wave’s exon skipping oligonucleotide WVE-N531 in 11 boys with exon 53 DMD. Biopsy data from eight of the boys showed a 6.4% mean muscle ...

Wave Life's WVE-N531 met all Phase 2 goals, showing exon skipping, dystrophin restoration. The therapy showed a 28.6% muscle fibrosis reduction, a 50% CK decline, and decreased IL-6/MCP-1 ...

PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, an established therapeutic target for approximately 13% of DMD patients, thereby aiming to restore the open reading frame and ...

The study achieved all its goals by demonstrating sustained and industry-leading exon skipping, muscle concentrations, and dystrophin restoration through 48 weeks, along with a 61-day tissue half ...

Global Duchenne Muscular Dystrophy Drugs Market value attained USD 3.9 Billion in 2025. The market will achieve USD 6.5 ...