Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …

Background When research evidence is limited, inconsistent, or absent, healthcare
decisions and policies need to be based on consensus among interested stakeholders. In …

Therapeutics for golodirsen to treat Duchenne muscular dystrophy (DMD) patients with
eligible mutations (www. drugs. com/newdrugs/fda-approves-vyondys-53 …

Molecular diagnosis for Duchenne and Becker muscular dystrophies (DMD/BMD) involves a
two‐tiered approach for detection of deletions/duplications using MLPA or array CGH …

Despite the early onset of clinical signs suggestive of Duchenne muscular dystrophy (DMD),
a diagnosis is often not made until four years of age or older, with a diagnostic delay of up to …

Abstract Duchenne Muscular Dystrophy (DMD) is a lethal progressive muscle-wasting
disease. New treatment strategies relying on DMD gene exon-skipping therapy have …

Introduction In this study we investigate associations between genotypic and
sociodemographic factors and the age of diagnosis of Duchenne muscular dystrophy …

Despite recent advances in the understanding of inherited muscle and neuromuscular
junction diseases, as well as the advent of a wide range of genetic tests, patients continue to …

Background Cardiomyopathy (CMP) is the most common cause of mortality in Duchenne
muscular dystrophy (DMD), though the age of onset and clinical progression vary. We …

Introduction Our objective was to identify recent CPGs for the diagnosis and management of
DMD and summarize their characteristics and reliability. Methods We conducted a scoping …