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

A coordinated, multidisciplinary approach to care is essential for optimum management of
the primary manifestations and secondary complications of Duchenne muscular dystrophy …

Importance Corticosteroids improve strength and function in boys with Duchenne muscular
dystrophy. However, there is uncertainty regarding the optimum regimen and dosage …

Objective To study vamorolone, a first-in-class steroidal anti-inflammatory drug, in Duchenne
muscular dystrophy (DMD). Methods An open-label, multiple-ascending-dose study of …

Glucocorticoids (GC) are an important risk factor for bone fragility in children with serious
illnesses, largely due to their direct adverse effects on skeletal metabolism. To better …

Objective We performed a prospective, cross-sectional analysis of neurodevelopmental
concerns and psychosocial adjustment in relation to DMD mutations in young steroid-naive …

The muscular dystrophies are a heterogeneous group of disorders defined by dystrophic
pathologic features on muscle biopsy. Clinically, they are characterized by progressive …

Deflazacort and prednisone/prednisolone are the current standard of care for patients with
Duchenne muscular dystrophy (DMD) based on evidence that they improve muscle strength …

Glucocorticoid steroids are widely used as immunomodulatory agents in acute and chronic
conditions. Glucocorticoid steroids such as prednisone and deflazacort are recommended …

Background Duchenne muscular dystrophy (DMD) is a fatal disease for which no cure is
available. Clinical trials have shown to be largely underpowered due to inter‐individual …