Caregiver-reported Patient Experiences with Duchenne Muscular Dystrophy: Qualitative In-trial Interviews 1 Year After Delandistrogene Moxeparvovec in the Pivotal EMBARK Trial.

ABSTRACT:

Introduction

Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease. The first approved DMD gene therapy, delandistrogene moxeparvovec, was evaluated in the phase 3, two-part, randomized, placebo-controlled EMBARK trial (NCT05096221), which did not meet statistical significance for its primary endpoint assessed at the end of Part 1 (Week 52). To gather patient experience data possibly not captured by traditional clinical outcome assessments, a qualitative in-trial interview substudy was conducted among caregivers (parents) (≥ 18 years) of male patients (≥ 4 to < 8 years with DMD) who completed Part 1 (Week 52) of EMBARK.

Methods

Semi-structured interviews, conducted with 23 parents within 14 days after the last EMBARK Part 1 visit, assessed DMD-related s

SUBMITTER: Audhya I 

PROVIDER: S-EPMC12804538 | biostudies-literature | 2026 Feb

REPOSITORIES: biostudies-literature