This study will evaluate ENTR-601-45, an investigational therapy for Duchenne muscular dystrophy or DMD, specifically targeting patients with mutations in the DMD gene amenable to exon 45 skipping.

tolerability and effectiveness of ENTR-601-45 in ambulatory patients with Duchenne who are exon 45 skipping amenable. Part A is a multiple ascending dose study designed to evaluate the safety ...

and research ethics committee to initiate a phase I/II study of ENTR-601-45 for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the DMD gene amenable to ...

Sarepta is running a phase 3 trial of golodirsen – as well as an exon 45-skipping drug called casimersen – that will also investigate muscle function outcomes and is due to generate results in ...

The early stage study will test ENTR-601-45, an experimental treatment for Duchenne muscular dystrophy in patients with a specific gene mutation that may respond to exon 45 skipping therapy.