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

Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal and cardiac muscle tissue to specifically skip exon 44 of the dystrophin gene and enable production of ...

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 ...

The company anticipates submitting a biologics licence application for accelerated approval in the US in early 2026.

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 DMD gene is made up of 79 exons, and mutations in that code can result in a dystrophin deficiency, which is responsible for the muscle wasting in DMD. Exon-skipping drugs are used to patch the ...

FORWARD-53 achieved all trial goals, demonstrating sustained and industry-leading exon skipping, muscle concentrations and dystrophin restoration through 48 weeks and a 61-day tissue half-life ...

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

FORWARD-53 achieved all trial goals, demonstrating sustained and industry-leading exon skipping, muscle concentrations and dystrophin restoration through 48 weeks and a 61-day tissue half-life ...

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 ...