Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in
2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved …

Background Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy
of childhood. Untreated, this incurable disease, which has an X‐linked recessive …

Introduction: ACE‐031 is a fusion protein of activin receptor type IIB and IgG1‐Fc, which
binds myostatin and related ligands. It aims to disrupt the inhibitory effect on muscle …

Contemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to
assess care recommendations and aid in planning future trials. Methods The Cooperative …

OBJECTIVES: The purpose of this study was to assess health-related quality of life (QoL) in
children with Duchenne muscular dystrophy (DMD), including development and field-testing …

Abstract Background and Purpose The aims of this study were to develop a clinical
assessment scale to measure functional ability in ambulant boys with Duchenne muscular …

Background This study aimed at analyzing the economic burden and disease-specific health-
related quality of life (HRQOL) of patients with spinal muscular atrophy (SMA) in Germany …

Aim To estimate health‐related quality of life (HRQOL) in patients with Duchenne muscular
dystrophy (DMD). Method HRQOL was assessed using the Health Utilities Index …

Objective To assess the feasibility, safety, and efficacy of intracoronary allogeneic
cardiosphere-derived cells (CAP-1002) in patients with Duchenne muscular dystrophy …

In this review we present an overview of quality of life (QOL) and QOL measures in
neuromuscular disorders. We discuss the characteristics of QOL measures used in …