October 2014

“It is exciting to see the big consortia put the genomic data of almost 400 patients together. This clearly highlights that by working together we can find new genes faster, helping us to explain what causes this often devastating disease in children.”- Tracy Dixon-Salazar, PhD

Pioneering T-Cell Therapy Leads to Remarkable Remission Rate


A landmark new study published in the New England Journal of Medicine shows that 90 percent of leukemia patients treated with a groundbreaking form of cellular therapy achieved complete responses. Led by The Children’s Hospital of Philadelphia’s Stephan M. Grupp, MD, PhD, the study is …

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International Study Pinpoints Childhood Epilepsy Genes


An international team of researchers recently identified gene mutations that can cause severe, difficult-to-treat forms of childhood epilepsy. Many of the mutations disrupt functioning in the synapse, the highly dynamic junction at which nerve cells communicate with one another. “This research represents a paradigm shift …

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Increases in Mitochondrial DNA Mutations Can Cause Shifts in Disease


Recent work by a mitochondrial medicine pioneer from The Children’s Hospital of Philadelphia details how subtle changes in mitochondrial function may cause a broad range of common metabolic and degenerative diseases. Mitochondria are tiny energy-producing structures within our cells that contain their own DNA. The …

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To Maintain Appropriate Antibiotic Use, Sustained Feedback Needed


A program that provides guidance to primary care physicians about appropriately prescribing antibiotics for children is effective, but its improvements wear off after regular auditing and feedback are discontinued. “Our findings suggest that interventions with outpatient healthcare providers should include continued feedback to clinicians to …

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CIRP Research Improves Teen Driving Training, Assessment


A recent article in the Wall Street Journal on “one of the most dreaded rites of child-rearing — teaching a teenager to drive,” notes recent research on teen driving and training can help teens learn to be better drivers and so avoid accidents. The article …

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Safety of Severe Asthma Care Outside the ICU Assessed


Clinicians at The Children’s Hospital of Philadelphia have extensive experience in treating children with acute asthma flares, partly due to the tremendous volume of patients with this respiratory condition who come from the urban community nearby. Asthma is one of the leading, serious, chronic illnesses …

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Rescuing Mutation May Operate in Bone Marrow Failure


Bone marrow failure syndromes are rare disorders in which the bone marrow does not produce enough blood cells, increasing the risk for life-threatening bleeding, anemia, and infections. While children with these conditions usually will need bone marrow transplantation, a small percentage experience spontaneous remission. Modern …

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Antibiotic Use in Infancy May Play Role in Obesity


While it is easy to blame the childhood obesity epidemic on too many french fries and video games, it is likely that multiple factors such as genetics and environment also contribute to excessive weight gain. A retrospective study based on data from The Children’s Hospital …

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Research Funding Shortfalls Require Personal Response


The Children’s Hospital of Philadelphia’s Peter M. Grollman, vice president of CHOP’s Office of Government Affairs, Community Relations, and Advocacy recently penned a powerful editorial on Philly.com arguing that tepid government support for medical researchers demands a personal response from voters. “You, too, may be …

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Children’s Hospital Researchers Honored


Four researchers from The Children’s Hospital of Philadelphia were recently honored with awards from the Perelman School of Medicine at the University of Pennsylvania. The 2014 Penn Medicine Awards of Excellence recognize faculty who exemplify the highest values of innovation, commitment to service, leadership, dedication …

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Core Seeks to Ease Recruitment Process


As the saying goes, it takes a village: without patients who believe in the promise that clinical research offers, medical studies can be doomed to fail. While every investigation is the product of hard work by teams of researchers, fellows, laboratory assistants, and coordinators, it …

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