Please provide your email address to receive an email when new articles are posted on . Ataluren preserved hand-to-mouth function in boys with nonsense mutation Duchenne muscular dystrophy. Ataluren ...

Involvement of different brain dystrophin isoforms was assessed in 3 groups of young males diagnosed with DMD. On their first assessment, 43 of the 70 patients with DMD had normal intelligence ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is linked ...

Please provide your email address to receive an email when new articles are posted on . “The adverse outcomes associated with GCs, including weight gain, appearance associated with Cushing syndrome, ...

Avidity Biosciences’ AOC 1020, newly dubbed del-brax, demonstrated a safe profile and was tied to functional improvement among people with facioscapulohumeral muscular dystrophy (FSHD) in a phase 1/2 ...

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated ...

Genetic testing, often via a blood sample, can confirm a muscular dystrophy diagnosis. It can also identify specific gene mutations to help guide treatment and determine whether or not someone is a ...

Doctors use physical therapy to treat Duchenne muscular dystrophy. It can help maintain muscle function, boost quality of life, and prevent complications. A physical therapist will tailor your ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the deterioration of skeletal muscle in the disease. The use of ...