Clinical Study Alert: Phase 2 Study of Brogidirsen in Boys with DMD

Researchers at NS Pharma are seeking boys with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping therapy to participate in a phase 2 clinical study (clinicaltrials.gov ID: NCT05996003) of their investigational therapy brogidirsen (NS-089/NCNP-02-201). DMD is caused by gene mutations that lead to the loss of the dystrophin protein, which is important for the structure and function of muscle cells. Brogidirsen is designed to target and promote skipping over a section of genetic code (exon 44) in order to avoid the gene mutation and produce more of the dystrophin protein. A phase 1/2 study (ID: NCT04129294) of this therapy has been completed and  it’s extension study (ID: NCT05135663) is currently in progress. are available under open access here.

The study

In the current phase 2 clinical study, the investigators are examining whether brogidirsen is safe and well-tolerated in people with DMD amenable to exon 44 skipping, and whether treatment may increase dystrophin levels and help stabilize or improve motor function.

Interested in participating?

Visit this link for more detailed information about this study. To learn more about the study or inquire about participation, contact the study coordinator at email: trialinfo@nspharma.com.