Gene Therapy :: Nationwide Children's Hospital

Gene Therapy Clinical Studies

Investigators with the Center for Gene Therapy currently are conducting numerous clinical research studies, described in detail below. Neurosciences at Nationwide Children's offers patients and families a comprehensive approach to the care for children and adolescents with disorders of the brain, spine and nervous system; from initial diagnosis and treatment to rehabilitation and long-term follow-up care.

Study to Evaluate Eteplirsen (Sarepta) in Early-Stage Duchenne Muscular Dystrophy (DMD)

Study to Evaluate Eteplirsen (Sarepta) in Older Children with Duchenne Muscular Dystrophy (DMD)

Follistatin Gene Therapy Study for Duchenne Muscular Dystrophy (DMD)

MicroDystrophin Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy (DMD)

Gene Transfer Clinical Trial for Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)

Phase 1 Gene Transfer Clinical Trial for Spinal Muscular Atrophy (SMA) Type 1

Research Study for Males 10 Years and Older with Duchenne Muscular Dystrophy

Phase 2 Trial of Corticosteroids for Infants and Young Boys with Duchenne Muscular Dystrophy

Prosensa Natural History Study (Pro DMD Study)

FOR Duchenne Muscular Dystrophy Study (FOR DMD Study)

Register for Gene Therapy Clinical Studies

If you are interested in registering for a clinical study for the Center for Gene Therapy, please fill out the form below and a coordinator will contact you shortly if you are eligible for a study.
Patient's Name (First and Last) *
Patient's Date of Birth (Month/Day/Year) *
Patient's Diagnosis and Type *
Your Name (First and Last) *
Your E-mail Address (If you do not have an email address, please enter NA.) *
Your Phone Number *
Your Country and State of Residence *
Your Child's Walking Ability: *
Steroid Use *


Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000