ELEVATE-44-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-44, was authorized by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee for the Clinical Trial of an Investigational Medicinal Product. The study aims to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the DMD gene that is amenable to exon 44 skipping.
What is ELEVATE-44-201 Clinical Study?
ELEVATE-44-201 is a global, two-part, randomized, double-blind placebo-controlled Phase 1/2 study evaluating the safety, tolerability and effectiveness of ENTR-601-44 in ambulatory patients with DMD who are exon 44 skipping amenable.