A National Leader in Pediatric Research

Family Foundations Fund Critical Study For Lysosomal Storage Disease MPS III

Three parent-run foundations have awarded investigators at Nationwide Children’s Hospital a $550,000 grant to support a natural history study of Sanfilippo Syndrome Types A & B.

Read more about this award.

Multi-Million Dollar Award from NINDS to Advance CNS-Directed Gene Therapy for Spinal Muscular Atrophy

Nationwide Children’s Hospital and Families of Spinal Muscular Atrophy announce the award of a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS) to advance a gene therapy development program for Spinal Muscular Atrophy (SMA).
 

Evidence Supports Blocking Immune Response to Enhance Viral Therapy against Solid Tumors

A new study, published in the March 12, 2013, issue of Molecular Therapy and led by Timothy Cripe, MD, PhD, chief, Division of Hematology/Oncology and Bone Marrow Transplantation at Nationwide Children’s, is shedding additional light on how viral therapy combined with a suppressed immune response could be more effective against solid tumors.
 

Scientists Studying Rare Diseases Quoted in Wall Street Journal Article

Center for Gene Therapy investigators Doug McCarty, PhD, and Kevin Flanigan, MD, were recently quoted in a Wall Street Journal article, “Families Push for New Ways to Research Rare Diseases.” Our researchers will soon launch a study related to the rare disorder, Sanfilippo Syndrome, a disease where the child is missing or has insufficient amounts of one of four enzymes needed to break down sugar molecules. Children with Sanfilippo Syndrome will ultimately lose their ability to walk, talk and eat.

Read the article here.
 

Five-year, $3 Million NIH Grant Awarded to Drs. Giannone and Bauer

Peter Giannone, MD, and John Bauer, PhD, principal investigators in the Center for Perinatal Research, have been awarded a five-year, $3 million grant from the Eunice Kennedy Shriver National Institute of Child Health and Human Development.

The project, titled “Indomethacin and Delayed Umbilical Cord Clamp for Preterm Infant Intraventricular Hemorrhage,” will investigate the clinical value and mechanistic effects of delaying umbilical cord clamping in extremely premature infants, particularly in relation to intracranial bleeding.
 

Variations Discovered: Study Reveals Need to Establish Best Practices for Diuretic Use in Preterm Infants with Bronchopulmonary Dysplasia

The study, published in the March 11, 2013 issue of Pediatrics, was led by Jonathan Slaughter, MD, MPH, an investigator in the Center for Perinatal Research and a neonatologist at Nationwide Children’s. He and his colleagues found significant variation in how infants receive these medications at hospitals across the nation.