Research News from CHOP
FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy
For decades, doctors and researchers have been working diligently to discover new treatments and provide more options for patients with Duchenne muscular dystrophy (DMD), a debilitating genetic disease that ravages the muscles in the body and leads to a loss of movement and coordination.
As Summer Heats Up, CHOP Researchers Study Caregiver Attitudes Toward Pediatric Vehicular Heatstroke
While those who were thought to put children at risk were viewed negatively, a large number of caregivers supported education efforts and technology use to reduce heatstroke risk.
CHOP Research Plays Major Role in New International Concussion Guidelines
Researchers from the Minds Matter Concussion Program at Children’s Hospital of Philadelphia have extensively researched the use of visio-vestibular examinations (VVEs) to improve concussion diagnosis in care. Now, that research is at the heart of new best practice recommendations made by a panel of more than 100 leading concussion experts around the world.
Self-Esteem of Kids with Short Stature Tied to Social Supports, Not Height
CHOP researchers have found that self-esteem in short youth is associated with coping skills and how supported they feel and not the degree of their short stature.
CHOP Researchers Identify Potential Target in Myeloid Malignancies
CHOP researchers have identified a molecule that plays a role in post-translational modification and activation in myeloid cancers, making it a potential therapeutic target.
CHOP Researchers Use Lab-Grown “Mini-Intestines” to Study Inner Lining of Gastrointestinal Tract for Crohn’s Disease Patients
Researchers from Children’s Hospital of Philadelphia (CHOP) confirmed that “mini-intestines” generated from patient tissue samples and grown in a lab can accurately replicate certain features of Crohn’s disease, which will allow them to study the epithelium, the thin layer of cells lining the intestine, that is broken down in patients with inflammatory bowel disease.
CHOP Researchers Develop Stable, Scalable Universal MHC Molecules
CHOP researchers have engineered stable, universal MHC-I molecules that can be produced rapidly at scale, allowing researchers to develop vaccines and immunotherapies more quickly.
CHOP Researchers Use “Deep Sequencing” to Identify Several Previously Undescribed Genetic Variants in Vascular Anomalies
The study captured genetic variants at extremely low levels, and dozens of patients began new therapies as a result of the findings.
First-of-its-Kind Open-Analysis Platform for Pediatric Brain Tumors Provides Robust Data Resource for Childhood Cancer Research
Collaboration among CHOP, Alex’s Lemonade Stand Foundation, the Pacific Pediatric Neuro-Oncology Consortium, and Children’s Brain Tumor Network paves the way for new collaborative models to accelerate discoveries.
American Academy of Pediatrics Develop National Standards for Neonatal Care
Dr. Eric Eichenwald, CHOP’s Division Chief of Neonatology, co-authored the first national standards for neonatal care published by the American Academy of Pediatrics (AAP).