• share
• favorite_border

A new study from Children’s Hospital of Philadelphia (CHOP) examined differences in the way girls and boys on the autism spectrum used certain types of words during storytelling. This study found that autistic girls used significantly more “cognitive process” words such as “think” and “know” than autistic boys, despite comparable autism symptom severity.

• share
• favorite_border

Researchers used CRISPR gene editing to thwart a lethal lung disease in an animal model in which a harmful mutation causes death within hours after birth. This proof-of-concept study showed that in utero editing could be a promising new approach for treating lung diseases before birth.

• share
• favorite_border

Hematology researchers have further refined how a treatment currently used urgently to control bleeding in hemophilia patients holds promise for prevention as well. A study in animals may lead to a new therapy for patients who now develop antibodies to the standard maintenance treatment.

• share
• favorite_border

Researchers from Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania analyzed the largest cohort to date of pediatric patients with high-risk neuroblastoma treated with proton radiation therapy, finding both that proton therapy was effective at reducing tumors and demonstrated minimal toxicity to surrounding organs.

• share
• favorite_border

New research recently published in the Journal of Pediatric Surgery found that post-operative success rates of pediatric thyroid patients, particularly children who require a thyroidectomy, correlate with the institution’s patient volume. The findings underscore how a highly experienced clinical team can make a big difference when it comes to patient outcomes.

• share
• favorite_border

Biomedical researchers plan to create the Pediatric Cell Atlas, a powerful new resource for fine-grained scientific understanding of human growth and development. Drawing on dramatic recent advances in technology, the Atlas will offer an unprecedented window into the unique biology of children by benchmarking healthy and abnormal tissues at the level of single cells—the basic units of biology.

• share
• favorite_border

For extremely preterm infants requiring resuscitation at birth, ventilation involving two sustained inflations, compared with standard intermittent positive pressure ventilation, did not reduce the risk of bronchopulmonary dysplasia or death at 36 weeks postmenstrual age.

• share
• favorite_border

A research team at Children’s Hospital of Philadelphia has developed an innovative computational tool offering researchers an efficient method for detecting the different ways RNA is pieced together (spliced) when copied from DNA.

• share
• favorite_border

Despite an FDA approval of growth hormone treatment for children with idiopathic short stature (ISS), the mean cost burden to patients and their families has increased over time.

• share
• favorite_border

Scientists have harnessed powerful data analysis tools and three-dimensional studies of genomic geography to implicate new risk genes for osteoporosis, the chronic bone-weakening condition that affects millions of people. Knowing the causative genes may later open the door to more effective treatments.

• share
• favorite_border

Physician-scientists in the Cancer Center at Children’s Hospital of Philadelphia have developed a preclinical, potent therapy attached to an antibody that targets a surface protein expressed in most childhood neuroblastomas, effectively killing cancer cells.

• share
• favorite_border

Mutations in MT-ATP6, which encodes an essential part of the mitochondrial motor known as ATP synthase that generates cellular energy, are much more variable than previously thought. This prompts the need to develop more precise clinical tests that can better determine the course of treatment for patients affected by mitochondrial disorder.

• share
• favorite_border

Automated programs can identify which sick infants in a neonatal intensive care unit have sepsis hours before clinicians recognize the life-threatening condition. A study team tested machine-learning models in a NICU population, drawing only on routinely collected data available in electronic health records.

• share
• favorite_border

New preclinical findings from extensive cell and animal studies suggest that cysteamine bitartrate, a drug already used for a rare kidney disease, could benefit patients with some mitochondrial disorders. No proven effective treatments yet exist for these complex conditions with severe energy deficiency

• share
• favorite_border

A pair of new studies has provided new insight into the challenges faced by children on the autism spectrum who exhibit symptoms of attention deficit/hyperactivity disorder (ADHD). According to the findings from researchers at Children’s Hospital of Philadelphia (CHOP), these children have difficulty with adaptive behavior, a key measure of independence.

• share
• favorite_border

Specialized lung cells appear in the developing fetus much earlier than scientists previously thought. Investigating the fetal signaling pathways active in the biological events by which alveoli form may offer future opportunities to treat lung damage caused by prematurity and other lung injuries.

• share
• favorite_border

A new map of newborn babies’ brains offers details of structure that will provide a new reference for researchers studying both typical brain development and neurological disorders. Using noninvasive, 20-minute MRI scans, researchers have revealed some of the complex and precisely organized brain architecture that emerges as the brain reshapes itself during the third trimester of pregnancy.

• share
• favorite_border

As part of a longstanding partnership with the UAE’s Ministry of Health and Prevention, Children’s Hospital of Philadelphia, a global leader in pediatric healthcare, exhibited some of its leading medical breakthroughs in the Ministry booth at the annual Arab Health Exhibition and Congress, in the Dubai International Convention and Exhibition Centre.

• share
• favorite_border

As the opioid epidemic continues to plague the United States, physician-researchers at Children’s Hospital of Philadelphia analyzed prescription patterns in children. They found that both duration of treatment and dose amounts declined between 2013 and 2017, while the rate of prescribing remained the same.

• share
• favorite_border

Over a year and a half after the successful separation of two infant twins joined at the top of their heads, surgical team leaders report on this dramatic case. The surgeons describe the innovative devices, elaborate planning and precisely orchestrated teamwork needed to perform the complex separation surgery.

• share
• favorite_border

Opioid drugs prescribed to children for pain relief after a typical pediatric orthopaedic procedure may be significantly overprescribed, according to a new study by researchers at Children’s Hospital of Philadelphia. The patients used less than 25 percent of the drugs, suggesting a potential risk of opioid diversion.

• share
• favorite_border

The Pennsylvania Pediatric Medical Device Consortium announces a partnership with two programs at the University of Pittsburgh. Formerly the Philadelphia Pediatric Medical Device Consortium, the PPDC’s new name reflects its statewide reach. The expansion follows a five-year $6 million grant renewal from the FDA.

• share
• favorite_border

Engaging in repetitive and ritualistic behaviors is part of typical child development. However, behaviors that develop into obsessive and compulsive symptoms (OCS) may represent a red flag for serious psychiatric conditions.

• share
• favorite_border

Researchers at Children’s Hospital of Philadelphia (CHOP) evaluated a digital medicine tool designed as an investigational treatment for children with autism spectrum disorder (ASD) and co-occurring attention/deficit-hyperactivity disorder (ADHD).

• share
• favorite_border

Pediatric oncologist and researcher Yael Mossé, MD, Director of the Neuroblastoma Development Therapeutics Program at Children’s Hospital of Philadelphia, has been named the inaugural holder of the Patricia Brophy Endowed Chair in Neuroblastoma Research.

• share
• favorite_border

Children’s Hospital of Philadelphia celebrates a pivotal moment in medicine: approval by the European Commission of LUXTURNA® (voretigene neparvovec), the first and only gene therapy for patients with an inherited retinal disease, last month. This also makes LUXTURNA the first gene therapy for a genetic disease that has received regulatory approval in both the U.S. and European Union.

• share
• favorite_border

Physician-scientists from Children’s Hospital of Philadelphia presented updated efficacy and safety data on Kymriah (tisagenlecleucel) --the first-ever FDA-approved personalized CAR T-cell gene immunotherapy for aggressive blood cancers, at the American Society of Hematology annual meeting, as well as first-of-its-kind research on overcoming CAR T-cell resistance.

• share
• favorite_border

Decades after two brothers died in childhood from a mitochondrial disease, scientists pinpointed the genetic cause as a founder mutation in an Ashkenazi Jewish ancestor. The discoverers say the causative gene should be included in prenatal genetic carrier screening tests.

• share
• favorite_border

As computational biologist Yi Xing, PhD, launches the Center for Computational and Genomic Medicine at Children’s Hospital of Philadelphia, he draws on his “bilingual” strengths. In this case, bilingual refers to his fluency in two scientific disciplines: computational biology and experimental biology.

• share
• favorite_border

An Oct. 31 event at Children’s Hospital of Philadelphia highlights the Philadelphia's role in manufacturing 21st century products: tools for precision medicine. The hospital's new Clinical Manufacturing Facility will produce clinical-grade vectors to deliver cellular and gene therapy for difficult-to-treat diseases.