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.
Tag Archive: rare disease
National Childhood Cancer Awareness Month is coming to a close, but new research endeavors are continuing the momentum and driving toward better treatment options.
Synapses were firing throughout the conference room in the Colket Translational Research Building as attendees at the 2017 Research Institute Scientific Symposium held May 2 learned about their colleagues’ intriguing research endeavors.
Researchers from the Center for Applied Genomics at The Children’s Hospital of Philadelphia investigating a serious, rare disease called common variable immunodeficiency have discovered a gene linked to immune defense.
A device created by a CHOP expert is changing the lives of children with a debilitating rare disease, offering hope of drastically improved outcomes.
As rare pediatric diseases go, Fibrodysplasia Ossificans Progressiva (FOP) is about as rare and debilitating as they come, affecting roughly one in two million people around the world.
Dr. Kalish recently received an award from the Alex’s Lemonade Stand Foundation to investigate tumor development associated with the rare childhood overgrowth disease Beckwith-Wiedemann Syndrome.
In the U.S., a disease is considered rare if it affects fewer than 200,000 people. Many rare diseases have genetic origins, and almost 70 percent of Americans affected by a rare disease are children, according to the National Organization for Rare Disorders (NORD).