Neurology Research and Innovation

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.

Tween Boy

Brain Characteristics Associated with Epilepsy and Internalizing Disorders

Diffuse alterations in brain structure and function are seen in youth with epilepsy and symptoms of anxiety or depression.

Two boys

A Natural History of Duchenne Muscular Dystrophy in Boys 3 to 6 Years Old

A prospective, multicenter study following 153 boys younger than 6 years old documents disease progression of Duchenne muscular dystrophy.

Heart Function

Cheaper, Generic Drug Just as Effective in Preserving Heart Function in Duchenne Muscular Dystrophy

A new clinical trial found a cheaper generic drug is effective in protecting the heart in boys with Duchenne muscular dystrophy.

Nationwide Children's Hospital Patient Photo

Targeting Cardiac-Related Strokes in Infants

New study describes risk factors for stroke in children with cardiac disease.

Pediatrics Nationwide

Nation’s First Clinical Trial for Pediatric Stroke Rehabilitation

A novel movement-based therapy is being evaluated in infants who suffered a stroke as newborns or in the womb.

spinal muscular atrophy SMA

FDA APPROVAL: Gene Therapy Comes of Age

In May, Zolgensma, a first-of-its-kind gene therapy for spinal muscular atrophy was approved.