Duchenne muscular dystrophy (DMD, OMIM 310200) is an incurable, X-linked recessive form of muscular dystrophy caused by mutations in the dystrophin gene ( DMD ) located on chromosome Xp21.2 [ 1 , 2 ]. The prevalence of DMD is less than 10 cases per …

Boys with Duchenne muscular dystrophy treated with givinostat versus placebo have less functional decline on the four-stair climb assessment.

Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular disorder caused by mutations in the gene encoding for dystrophin, a protein expressed in the sarcolemma of the skeletal and cardiac muscles. Lack of dystrophin affects the …

Duchenne muscular dystrophy (DMD) is a genetic disease that causes muscle weakness and wasting. Children born with DMD have a mutation in the dystrophin gene [ 1 ]. The dystrophin gene is made up of 79 exons coding for a protein of 3,685 amino acid …

Duchenne muscular dystrophy (DMD) is a recessive, X-linked inherited neuromuscular disorder that affects 1 in 3500–5000 newborn males worldwide [ 1 – 4 ] and <1 in 1,000,000 newborn females [ 5 ]. It is characterised by a severe deficiency or …

Duchenne muscular dystrophy (DMD) is an X-linked, recessive, degenerative neuromuscular disorder that affects 1 in 3500–5000 male births worldwide [ 1 – 3 ], and has a pooled global prevalence of 7.1 cases per 100,000 males and 2.8 cases per …

Duchenne muscular dystrophy is a devastating disease that leads to progressive muscle loss and premature death. While medical management focuses mostly on symptomatic treatment, decades of research have resulted in first therapeutics able to …

Duchenne Muscular Hystrophy (DMD) is a X-linked inherited genetic disease caused by mutations in the DMD gene. Variants leading to loss of dystrophin function, a product of this gene, modify the structure of the muscle cell that is progressively …

Duchenne muscular dystrophy (DMD) is a rare X-linked condition presenting in early childhood with estimates of 1 in 3,500 live male births affected [ 1 ]. The use of long-term oral glucocorticoid as a disease modifier has been adopted as standard …

Over the past three decades, patient-centered research has emerged as critically important for understanding the impact of treatments on key stakeholders [ 1 , 2 ]. Patients have become not only central to outcomes measurement for new treatments [ …

Duchenne muscular dystrophy (DMD) is an X-linked inherited myopathy that causes progressive skeletal and cardiac muscle disease. DMD's critical features are chronic cardiac muscle inflammation and subsequent fibrotic tissue deposition. Heart …

Duchenne muscular dystrophy (DMD) is an X-linked, incurable, degenerative neuromuscular disease caused by mutations in the DMD gene coding for dystrophin protein. The absence of dystrophin compromises the integrity of the sarcolemma and leads to …

Public health surveillance, defined as the "systematic and continuous collection, analysis, and interpretation of data" [ 1 ] is foundational to public health practice [ 2 ]. Public health surveillance provides accurate, representative information …

Duchenne muscular dystrophy (DMD) is a severe X-linked disease characterized by progressive muscle weakness [ 1 , 2 ]. Treatment of DMD is always a clinical challenge. The pathology of DMD involves gene abnormality, oxidative stress [ 3 – 5 ].