Neurology Research and Innovation

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.

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.

Limb-Girdle Muscular Dystrophy Type 2E

Clinical Trial Open for Patients With Limb-Girdle Muscular Dystrophy Type 2E

Researchers are investigating the safety and effectiveness of a new gene therapy product that replaces the defective β-sarcoglycan gene.

Kevin M. Flanigan, MD

Whole Exome Sequencing Reveals Ultra-Rare Neuromuscular Disorders

Clinical use of large-scale genetic screening helped end the diagnostic odyssey for many patients with nonspecific neuromuscular phenotypes.

Duchenne Muscular Dystrophy

DNA Variations Associated With Prolonged Walking in Duchenne Muscular Dystrophy

Genome-wide association study identifies two variants that may yield therapies for children with DMD.

Suicide Prevention

Concussion is Associated With a 2x Higher Risk of Suicide

A meta-analysis shows people who have experienced concussion and/or mild traumatic brain injury are more likely to attempt suicide and to have suicidal ideation than people who have not experienced the injuries.

Enzyme Treatment Slows Decline in Common Form of Batten Disease

Study also indicates early treatment may provide best outcomes.