September 2014

“CHOP is at the forefront of teen driver safety research, and it is an amazing place to take these leaps because of our interdisciplinary nature."- Flaura Koplin Winston, MD, PhD

Breakthrough Work Leads to Rare Bone Disease Trial


As rare pediatric diseases go, Fibrodysplasia Ossificans Progressiva (FOP) is about as rare and debilitating as rare diseases come. Affecting approximately one in two million people around the world, FOP is a condition in which ectopic (extraskeletal) bone is formed when “muscle tissue and connective …

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Mutation Helps Explain How Our Body Clock is Set


Most of us have failed to get enough sleep on a few occasions. Maybe it was as a college student studying for final exams, or as a new parent consoling an infant who is teething. Remember how you dragged through the next day in a …

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Investigators Take Clinical Look at Teen Driver Safety


Crashes remain the leading cause of young adult death (ages 16 to 24) with four times the number of deaths from cancer and 38 times the number of deaths from the flu. In fact, a teen’s highest lifetime risk of crashing occurs immediately following the …

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Discovering, Optimizing Epilepsy Therapies with Genetics


A new study led by The Children’s Hospital of Philadelphia’s David Bearden, MD, and Ethan M. Goldberg, MD, PhD, supports the idea that the identification of specific genetic targets could lead to a sea change in the way epilepsy is treated. Published in the Annals …

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Intervention Aims to Promote Effective Practice for ADHD


Approximately 7,800 children with attention-deficit/hyperactivity disorder (ADHD) visit primary care practices across The Children’s Hospital of Philadelphia Care Network. Treating such a large patient population that is coping with a multifaceted, chronic condition can be demanding for busy primary care physicians (PCPs). That is why …

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Healthy Weight Program Launches Study on Microbiome


Trillions of naturally occurring bacteria and other microbes coexist in the human gut and are part of how we process food and harness energy. Known as the microbiome, this complex collection’s potential role in early weight gain and obesity has become a fascinating area of …

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Report Supports Prenatal Spina Bifida Surgery as Standard of Care


Reporting on 100 recent cases of fetal surgery for spina bifida, specialists from The Children’s Hospital of Philadelphia achieved results similar to those in a landmark clinical trial that established a new standard of care for prenatal repair of this birth defect. The single-center study …

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Researchers Discover Gene Mutation in Blood Disorder


An international team of scientists has identified a gene mutation that causes aplastic anemia, a serious blood disorder in which the bone marrow fails to produce normal amounts of blood cells. Studying a family in which three generations had blood disorders, the researchers discovered a …

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Genetic Study Gives Clues to Intellectual Abilities


As students with bulging backpacks return to school this fall, each one also brings a unique skill set to the classroom. One child may be a math whiz, while their buddy in the next desk is an avid reader. A large genetic study conducted by …

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Gene-regulating Protein Reaches Multiple Disease Pathways


Genomic scientists at The Children’s Hospital of Philadelphia recently found strong evidence that the protein family FOXA2 is a master regulator of genetically vulnerable pathways in multiple diseases. Study leader Struan F.A. Grant, PhD, holder of the Daniel B. Burke Chair for Diabetes Research at …

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Scholar Hope Grant Supports Neuroblastoma Research


Pediatric oncology researchers at The Children’s Hospital of Philadelphia are driven to find new treatments for childhood cancer, and the Hyundai Hope On Wheels program is supporting their hard work by awarding a 2014 Scholar Hope grant to help fund investigations that focus on the …

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Research Institute Leader Named to Brain Tumor Foundation Panel


The Children’s Hospital of Philadelphia Research Institute’s Tom Curran, PhD, FRS, was recently named to a panel of experts who will advise the Pediatric Brain Tumor Foundation (PBTF). A pediatric brain tumor expert and Deputy Scientific Director of the Research Institute, Dr. Curran was one …

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