Study shows long-term safety of golodirsen

Data from a study of godolirsen has demonstrated the long-term safety of the treatment and showed increased production of dystrophin in the muscles of study participants.

The research is a collaboration between a number of academic researchers and Sarepta Therapeutics, the company that makes the drug. They have published data from their phase 1/2 study of golodirsen, a drug that may be used for the treatment of boys with Duchenne muscular dystrophy who have a confirmed mutation amenable to exon 53 skipping.

The study was not designed to demonstrate the clinical efficacy of the drug, and the number of participants and controls were very small. But an analysis of the data suggested that people with Duchenne muscular dystrophy may gain some functional benefit from treatment with golodirsen, which may still be observed two years after starting treatment.

This study supports the evaluation of golodirsen, which is currently undergoing a Phase 3 trial (NCT02500381).