Eteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative Patient Experience Study.

ABSTRACT:

Introduction

Duchenne muscular dystrophy (DMD) is characterized by rapid functional decline. Current available treatment options aim to delay disease progression or stabilize physical function. To aid in healthcare providers' understanding of the symptoms of disease that impact patients' experience, this study explored children's physical functioning, activities of daily living (ADLs), and health-related quality of life (HRQoL) after receiving eteplirsen, a weekly infusion indicated for individuals with DMD with exon 51 skip-amenable mutations.

Methods

Fifteen caregivers of male individuals with DMD participated in a 60-min, semi-structured interview. Open-ended questioning explored changes in the children's condition or maintenance in abilities since eteplirsen initiation.<

SUBMITTER: Iff J 

PROVIDER: S-EPMC11263411 | biostudies-literature | 2024 Aug

REPOSITORIES: biostudies-literature