Bundled up in winter clothes from head to toe, 7-year-old Eric rolls a snowball around the backyard with his older brother, building it into a sizeable snowman. Mom, watching from her perch at the kitchen window, can hear her boys laughing — until — Eric doubles over in acute chest pain, crying out for her. Rushing to his side, she wonders how many more of these excruciating episodes and trips to the hospital Eric can endure.
Tag Archive: sickle cell disease
It’s still a month before teachers and students are officially back to school, but here at Children’s Hospital of Philadelphia Research Institute, you can learn something new every day. In this edition of our biweekly news roundup, discover the latest findings from our ophthalmologists on how clinicians should choose to screen premature babies for a potentially blinding eye disorder, find out how CRISPR-based technology allowed scientists to reveal insights into sickle cell disease, and prepare for an educational and exciting speech from the recently announced keynote speaker at PolicyLab’s upcoming 10th Anniversary Forum
The year 2017 might be coming to a close, but research continues to ramp up at Children’s Hospital of Philadelphia, with exciting developments in the fields of brain science, hemophilia, gene therapy, and more. In this week’s roundup of headlines, we take a look at remarkable reports from CHOP and Penn Medicine about the brain’s ability to reorganize itself after limb amputation, the first U.S. effort to observe the use of medical cannabis for children with autism, and exciting innovations to improve sickle cell disease treatment presented at the 59th American Society of Hematology Annual Meeting and Exposition. Read on to discover more about these brilliant breakthroughs.
Notable awards, new autism initiatives, and a novel approach to managing sickle cell disease are all part of this week’s roundup of research news.
Investigators at The Children’s Hospital of Philadelphia are exploring a new gene therapy approach that aims to reactivate the production of fetal hemoglobin as a potential intervention for patients with sickle cell disease.
Kelly A. Dougherty, PhD recently received an award from the NIH that will support her investigation of vitamin A supplementation and sickle cell disease.
Hematologists have long sought to reactivate fetal hemoglobin as a treatment for children and adults with sickle cell disease (SCD). Researchers at CHOP have manipulated key biological events in adult blood cells to produce a form of hemoglobin normally absent after the newborn period.
Sickle cell disease affects millions of people worldwide and is the most common inherited disorder in African Americans, of whom 1 in 375 is born with the disease.
In a recent interview with the National Initiative for Children’s Healthcare Quality (NICHQ), Kwaku Ohene-Frempong, MD, Doctor Emeritus of CHOP’s Comprehensive Sickle Cell Center, discussed his work with sickle cell disease.