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

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

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 ...

Little Jamie Tierney Jr's parents have been desperately trying to save him since he was diagnosed in March 2022.

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 ...

Avidity Biosciences (RNA) announced del-zota topline data from the Phase 1/2 EXPLORE44 trial in people living with Duchenne muscular dystrophy amenable to exon 44 skipping demonstrating consistent ...

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 ...

Parents Jamie, 33, and Bobbie, 32, have spent the last three years desperately seeking treatments abroad and have been ...