Advancing Care of Neurological Conditions Through Research

University Hospitals Rainbow Babies & Children’s neurology team stays on the leading edge of pediatric neurological advancements through medical research and participating in clinical trials and studies. As part of a large academic medical center, we are constantly seeking new, innovative and better ways to diagnose and treat our patients for improved quality of life.

Gene Replacement Therapy for Spinal Muscular Atrophy and Neuromuscular Conditions

Gene replacement therapy – essentially, replacing a defective gene – is proving effective for genetic neuromuscular conditions such as spinal muscular atrophy (SMA). University Hospitals is an established leader in gene replacement therapy, and UH Rainbow Babies & Children’s is on the forefront of administering gene therapy treatments in pediatric patients.

Clinical Trials for Treating Rare Forms of Epilepsy

Dravet Syndrome is a rare form of epilepsy. UH Rainbow Babies & Children’s is currently participating in a clinical trial of a new medication that has the potential of controlling seizures in children with Dravet Syndrome, which could be life-changing for these patients.

Starting in late 2018, we will be participating in a clinical trial for another medication to treat Lennox-Gastaut Syndrome, also a rare and genetic form of epilepsy where seizures currently cannot be controlled by medications.