Early data suggests delpacibart zotadiersen may slow down DMD

11 October 2025

A new medicine called delpacibart zotadiersen (or del-zota for short) might help slow down Duchenne muscular dystrophy (DMD).

People with DMD have a change in their genes that stops their bodies from making dystrophin, a protein that helps protect muscles. Del-zota is a type of treatment called exon-skipping. This means it hides a small part of the gene (called exon 44) so the body can make a shorter version of dystrophin that still works.

In the study, 23 people with DMD aged between 7 and 27 took part. Some could still walk, and some could not. After taking del-zota for around 5 months, on average, they made 25% more dystrophin than usual.

17 participants continued to receive the treatment and have been followed for around one year. During that time, the treatment helped lower levels of creatine kinase – a substance in the blood that shows muscle damage. On average, levels dropped by 80%. About half of the participants also had levels in the normal range after a year.

The study also showed that people who took del-zota did better in movement tests, like walking 10 metres or climbing stairs. These results were compared to natural history data, which shows how DMD usually changes over time without treatment.

This data is encouraging, but the study was small. A bigger trial is now needed to check if del-zota really helps people with DMD and is safe. In particular, future studies will need to compare del-zota to a placebo (dummy drug) to confirm whether the improvements in movement are truly due to the treatment.