June 2014

“We want our kids participating in sports. I’d be much more troubled if young people were shying away from sports. As parents, though, we want to keep them safe, and that means we have to have better information.”- President Barack Obama

Gene Study Points to Novel Pathway for Diabetes Treatment


New research from The Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania details how a diabetes-related gene functions on a biological pathway that affects the release of insulin. Finding drugs that act on that pathway may eventually lead to a new treatment for type 1 diabetes. “In 2007, our genomics team found the first gene in a genome-wide  …

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Using Children’s Feedback to Measure, Respond to Adversity


As any pediatrician will tell you, children are not just little adults. Assessing and treating pediatric health issues is not the same as assessing and treating adult health issues, only in miniature. Children are different from adults in myriad ways — physically, developmentally, and emotionally. They also use language differently than adults do. For one, children have much smaller vocabularies t …

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Platelet Granule Formation Gives Insight Into Rare Disease


If you take a normal platelet and examine it under an electron microscope, you will see a bunch of black dots. The specks may not seem like much at first, but cell biologists at The Children’s Hospital of Philadelphia Research Institute speculate that these dense granules hold the key to unlocking the mechanisms behind a rare disease called Hermansky-Pudlak syndrome (HPS) and other forms of bleedi …

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Gene Networks Could Offer Autism Treatment Targets


After analyzing the DNA from thousands of patients, investigators from Children’s Hospital have uncovered several gene networks that may play important roles in autism. These networks may offer attractive targets for developing new autism drugs or repurposing existing drugs that act on components of the networks. Hakon Hakonarson, MD, PhD, director of the Center for Applied Genomics, and his team  …

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Concussion Prevention, Care Are Key Targets for Research


Few healthcare issues reach the presidential podium and ESPN headlines, but the topic of sports-related concussions in youth scored both, therefore gaining momentum as a national priority. President Obama hosted the first-ever White House Healthy Kids & Safe Sports Concussion Summit on May 29, where key stakeholders gathered to promote new efforts that will increase research to expand knowledg …

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Biomarkers for Mitochondrial Diseases Emerging


A major limitation in many therapeutic interventions is the inability to follow how they affect the early biochemical steps of chronic disease. In some cases, patients and clinicians spend months tracking symptoms to figure out if a treatment is working. A new approach being developed at The Children’s Hospital of Philadelphia and the University of Pennsylvania could allow investigators to know wi …

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Study Spurs Insights Into Chronic Kidney Disease


Identifying the best strategies to slow the progression to kidney failure for children with chronic kidney disease (CKD) is a constant challenge for clinicians. They must finely tune their treatment plans and judiciously anticipate when to begin dialysis or place patients on a kidney transplant waiting list. The Chronic Kidney Disease in Children Study (CKiD) is a prospective cohort study that was …

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New Center of Emphasis to Support Pharmacologic Research


Established to speed research from the bench to the bedside, The Children’s Hospital of Philadelphia Research Institute’s Centers of Emphasis concentrate investigator efforts and resources. Bringing together many of the most pioneering researchers in the country, CHOP Research’s Centers of Emphasis are well known for their consistently excellent, forward-thinking investigations. The Research Insti …

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Website Offers Expert Injury Tips in Spanish


A growing number of Spanish-speaking families in the United States look for health and parenting information online. Each year 8 million children – including nearly 1 million Hispanic children –experience an injury that requires emergency room care or a hospital stay. A team of experts at The Children’s Hospital of Philadelphia launched a new Spanish-language website, AfterTheInjury.org/es, to hel …

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CHOP Research CSO Named to PA Bio Board


In an example of how many Children’s Hospital investigators contribute their time and talents to social and professional organizations, Chief Scientific Officer and Executive Vice President of The Children’s Hospital of Philadelphia Research Institute Philip R. Johnson, MD, was recently named to Pennsylvania Bio’s Board of Directors. Devoted to ensuring Pennsylvania “is the global leader in the bi …

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