DUCHENNE MUSCULAR DYSTROPHY (DMD) is a severe genetic condition characterized
by progressive muscle degeneration and weakness due to the absence of the dystrophin …

Duchenne muscular dystrophy is a severe X-linked disease caused by mutations in the
DMD gene that lead to nearly complete loss of dystrophin in skeletal and cardiac muscle …

Using an animal model of Duchenne muscular dystrophy (DMD), investigators were able to
activate thyroid-stimulating hormone receptor (TSHR), which is highly expressed in …

The 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held
March 19-22, in Dallas, Texas, features a range of preclinical, translational, and clinical …

Duchenne muscular dystrophy (DMD) is experiencing its renaissance. After being the first
muscular dystrophy in which the disease gene has been identified, it is now the first …

Recently, the FDA granted orphan drug designation to Avidity Biosciences' AOC 1044, an
investigational therapy in development for the treatment of Duchenne muscular dystrophy …

A new paper outlines protocols for administering and monitoring the gene therapy—
delandistrogene moxeparvovc—for Duchenne muscular dystrophy, including gene testing to …

I read with interest the guidelines on corticosteroid treatment in Duchenne muscular
dystrophy (DMD). 1 As a pediatrician, I find 0.75 mg/kg/d of prednisone very high. I …

The Mind Moments® podcast features exclusive interviews with leaders in the field
discussing the latest research and disease management strategies across the breadth of …

" The significance of disease awareness cannot be overstated. It is crucial for clinicians in
the neuromuscular field to be informed about what interventions are possible from a public …