The muscular dystrophies are rare orphan diseases, characterized by progressive muscle
weakness: the most common and well known is Duchenne muscular dystrophy which affects …

There is an unmet need to identify biomarkers sensitive to change in rare, slowly
progressive neuromuscular diseases. Quantitative magnetic resonance imaging (MRI) of …

Neuromuscular diseases are characterized by progressive muscle degeneration and
muscle weakness resulting in functional disabilities. While each of these diseases is …

Late onset Pompe disease (LOPD) is a slow, progressive disorder characterized by skeletal
and respiratory muscle weakness. Enzyme replacement therapy (ERT) slows down the …

Objective No treatments are approved for Becker muscular dystrophy (BMD). This study
investigated the efficacy and safety of givinostat, a histone deacetylase pan-inhibitor, in …

More than 100 attendees from Australia, Austria, Belgium, Canada, China, Germany, Hong
Kong, Indonesia, Japan, Malaysia, the Netherlands, the Philippines, Republic of Korea …

Objective Limb girdle muscular dystrophy type R9 (LGMD R9) is an autosomal recessive
muscle disease for which there is currently no causative treatment. The development of …

Introduction The purpose of this study was to present and compare pain between adult
males with Duchenne (DMD), Becker's (BMD), Limb-Girdle (LGMD) Facioscapulohumeral …

Quantitative MRI and MRS of muscle are increasingly being used to measure individual
pathophysiological processes in Becker muscular dystrophy (BMD). In particular, muscle fat …

The latest results obtained from the study of long cohorts of patients with various
neuromuscular diseases open the door to the use of this technology in clinical trials, which …