Preferences for gene therapy in Duchenne muscular dystrophy: insights from patient and caregiver interviews and attribute development.

ABSTRACT:

Background

Duchenne muscular dystrophy (DMD) is an X-linked degenerative muscle disease with no curative treatment available to date. The current long-term use of corticosteroids is associated with severe adverse effects. With the progress of promising gene therapy for DMD, this research aims to identify the key characteristics that matter most to patients, develop attributes for a subsequent quantitative preference study, ultimately aimed to inform future market access and clinical decision-making on gene therapy.

Methods

A literature review was conducted, followed by semi-structured interviews with DMD patients and caregivers to explore their preferences regarding DMD treatment benefits and side effects and gene therapy as a promising treatment option. A ranking exercise h

SUBMITTER: Desmet T 

PROVIDER: S-EPMC12515650 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature