Encouraging First Report of Systemic Delivery of Micro-dystrophin Gene Therapy in Children With DMD
Researchers have published results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with Duchenne muscular dystrophy. Initial findings suggest that the therapy can provide functional improvement that is greater than that observed under the standard of care.
Diffuse alterations in brain structure and function are seen in youth with epilepsy and symptoms of anxiety or depression.
A prospective, multicenter study following 153 boys younger than 6 years old documents disease progression of Duchenne muscular dystrophy.
A new clinical trial found a cheaper generic drug is effective in protecting the heart in boys with Duchenne muscular dystrophy.
New study describes risk factors for stroke in children with cardiac disease.
A novel movement-based therapy is being evaluated in infants who suffered a stroke as newborns or in the womb.
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