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Nationwide Children’s Hospital Selected as Clinical Coordinating Center in Large National Clinical Trial Sponsored by National Heart Lung and Blood Institute (NHLBI)
The National Heart Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) has announced that Nationwide Children’s Hospital has been selected for funding as the Clinical Coordinating Center for the multicenter randomized trial: “Percutaneous intervention versus observational trial of arterial ductus in lower gestational age infants (PIVOTAL)”.
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Investigators Develop Technique to Effectively Edit NK Cells to Target Specific Cancer Cells
Originally, NK cells seemed promising for a natural therapy to fight cancer due to their innate ability to recognize any cells expressing stress or signs of infection. But investigators quickly realized they faced three key challenges: first, it was difficult to grow a large enough number of NK cells in the lab to offer useful infusions at a reasonable cost; second, an ideal universal donor solution was needed to make NK cell products faster, more reliably and with off-the-shelf availability; and third, NK cells were resistant to genetic modifications that might help them better target specific types of cancer cells. Nationwide Children’s Hospital investigators and their collaborators painstakingly solved the first two problems over the past decade. Now, they’ve conquered the third.
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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.
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Nationwide Children’s Hospital Columbus Marathon & 1/2 Marathon Announce 2022 Patient Champions
The Nationwide Children’s Hospital Columbus Marathon & 1/2 Marathon is pleased to announce the 2022 “Patient Champions” who will represent 24 of the 26 miles on the course. The other two miles, the Encore Mile, will honor former Marathon Patient Champions, and the Angel Mile, will celebrate the lives of those who are no longer with us.
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Catherine Krawczeski, MD, Named Chief of the Department of Pediatrics at Nationwide Children’s Hospital
Catherine Dent Krawczeski, MD, has been named Physician-in-Chief and Chief of the Department of Pediatrics at Nationwide Children’s Hospital. Dr. Krawczeski will also serve as Chair of Pediatrics at The Ohio State University College of Medicine, pending approval from The Ohio State University Board of Trustees. Dr. Krawczeski will begin this new role in Summer 2022.
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A Single-Dose Cancer Immunotherapy via Gene Transfer
Researchers at Nationwide Children’s Hospital report proof-of-principle results for a new gene therapy cancer treatment. The off-the-shelf, single-dose immunotherapy serves as an alternative to CAR-T therapy and can be engineered to be on-demand.
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Epilepsy Centers in United States Expand in Size and Specialized Testing Services with Accreditation
New research from the Abigail Wexner Research Institute at Nationwide Children’s Hospital has found that as a result of the increased number of accredited epilepsy centers in the United States, drug-resistant epilepsy (DRE) patients have easier access to specialized testing and services. The report, which describes level 3 and level 4 epilepsy centers that were accredited by the National Association of Epilepsy Centers (NAEC) from 2012 to 2019, published this week in Neurology.
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On Our Sleeves® Survey Reveals Most Parents Believe Their Children Will Have Mental Health-Related Challenges Going Back to School
Every new school year comes with its own set of emotions – from worry to excitement – for kids. After years of a global pandemic, safety concerns and other stresses of today, we have to prioritize checking in with children to understand what they are thinking and feeling.
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The Great Collide: The Impact of Children’s Mental Health On the Workforce
The pediatric and adolescent mental health crisis –brought to new attention and made more urgent by the stresses of the pandemic – is now showing to have direct and dramatic implications in the workplace. On Our Sleeves, the movement for children’s mental health, recently conducted a first-of-its-kind national study in spring 2021, funded by the Nationwide Foundation, to specifically evaluate the impact of children’s mental health on parents' work performance and, in turn, on companies’ success.
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New Disorder of Copper Metabolism Identified, Caused by Variants in CTR1 Gene
Copper is essential for many cellular functions, including cellular respiration, antioxidant defense, neurotransmitter biosynthesis and neuropeptide amidation, among others. Until recently, only two inborn errors of copper metabolism were well established. Both are rare. Wilson's disease and Menkes disease result from mutations in the copper-transporting P-type ATPases; ATP7B and ATP7A, respectively.