Sarepta has announced they are recruiting for their MIS51ON clinical research study in the UK.
A randomized, double-blind, dose finding and comparison study of the safety and efficacy of a high dose of Eteplirsen, preceded by an open-label dose escalation, in patients with Duchenne muscular dystrophy with deletion mutations amenable to Exon 51 skipping.
About the study
The study is researching the investigational use of a drug called eteplirsen, a potential treatment which is designed to target Duchenne gene mutations amenable to exon 51 skipping. This study is seeking to determine if a higher dose of eteplirsen is also safe and/or effective in treating Duchenne.
For more information on the study, including additional inclusion/exclusion criteria and a current list of participating global study centres, visit clinicaltrials.gov and search “NCT03992430” or email Sarepta directly to find out more about UK recruitment SareptAlly@Sarepta.com.
Interesting further information
- Visit Duchenne explained to understand the terminology used when we talk about Duchenne research
- Learn more about the science of Duchenne at the Action Duchenne International Conference, keep informed by signing up to receive news and updates from Action Duchenne here;
