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Early Phase Gene Therapy Study for SMARD1/CMT2S Disease Now Enrolling
Nationwide Children's Hospital has launched a new Phase I/IIa gene replacement clinical study to evaluate the safety of a gene therapy for IGHMBP2-related diseases, specifically spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S). SMARD1 and CMT2S are rare inherited conditions that are both caused by mutations in the IGHMBP2 gene, of which more than 60 have been found to date. SMARD1 is the most severe form of these diseases and causes muscle weakness and respiratory failure in infancy.
News
Bellville, Ohio, Teen and Family Travel to Capitol Hill to Advocate for Pediatric Cancer Research
The Reed family of Bellville, Ohio, is taking their story to Capitol Hill to deliver an important message to their members of Congress. Nationwide Children’s Hospital patient, Grant Reed, 15, and his family will join more than 40 other pediatric patients and their families to meet with
News
National Study Finds Rising Rate of Marijuana Exposure Among Children 5 Years Old and Younger
Debates about legalizing marijuana have focused on crime rates, economic benefits, and health effects among adults. But a study published today from researchers at Nationwide Children’s Hospital shows that the risk to young children of swallowing, breathing in or otherwise being exposed to
Article
Computational Genomics
The “big data” nature of genome-based clinical tests and research studies requires advanced high-performance computing and bioinformatics platforms to rapidly analyze and interpret information.
Article
Research and Innovation
Our current clinical research includes assessment of surgical outcomes, the Americleft Outcomes Project and studies investigating early speech-language development in infants with cleft palate.
News
Sophias Cure Foundation Donates $650,000 in 2012 to Nationwide Childrens Hospital
Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, along with a team of Spinal Muscular Atrophy (SMA) researchers and clinicians, received $650,000 in grants in 2012 from Sophia’s Cure Foundation for SMA
News
Grand Challenge Grant Awarded to Team Led by Nationwide Childrens Researcher
The Saving Lives at Birth: A Grand Challenge for Development recently shortlisted 53 finalists from around the world from more than 400 entries. Only 22 teams were awarded grants for their bold, new ideas to save the lives of mothers and newborns in developing countries.
Article
Cheerleading Safety
Many cheerleaders are injured while practicing and performing each year in the US. With proper training, rules and spotting, however, many of these injuries can be prevented.
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Gene Therapy Center of Excellence
The Gene Therapy Center of Excellence (GTCOE) at Nationwide Children's works with patients with rare and life-limiting diseases who could be treated with gene therapy. The center offers a full range of support for patients and families.
Article
Batteries
There are two types of batteries. One kind is long and skinny like AA or C. Many flashlights and toys use that kind. The other kind of battery is round, flat, and looks like a silver button. Button batteries are in watches, key fobs, thermometers, singing cards, and many other items in your home.