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

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 ...

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

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 ...

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

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

The EXPLORE44 study enrolled DMD patients with DMD mutations amenable to exon 44 skipping (DMD44), a group that accounts for around 6% of all DMD cases in the US. It showed a statistically ...

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 ...

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 ...

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 ...