The APS will recognize its new members at its Presidential Plenary at the Pediatric Academic Societies 2025 Meeting in April. Current members nominate new members by recognizing individuals who have distinguished themselves as child health leaders, clinicians, policymakers, researchers, scholars and teachers.
Researchers at Children鈥檚 Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy trial for Danon disease (DD), a rare, X-linked heart condition caused by a single gene mutation. The phase 1 trial was a collaboration between CHOP and the University of California, San Diego Medical Center, the University of Colorado, Anschutz Medical Center, Boston Children's Hospital and Rocket Pharmaceuticals. The data on the results of the RP-A501 Phase 1 trial, presented at a late breaking session today at the American Heart Association Scientific Sessions 2024 in Chicago, were also published in the New England Journal of Medicine.
A multi-institutional program led by researchers at the Center for Data Driven Discovery in Biomedicine (D3B) at Children鈥檚 Hospital of Philadelphia (CHOP) has received up to $10 million Advanced Research Projects Agency for Health (APRA-H) Mission Office Innovative Solutions Openings award to research, validate and implement a new model for sharing and integrating data at a national level.
Researchers at Children鈥檚 Hospital of Philadelphia (CHOP) revealed for the first time that children, adolescents and young adults may experience very rare neurological issues of paraparesis and quadriparesis following chimeric antigen receptor T-cell (CAR-T) therapy, a type of immunotherapy used to treat B-cell Acute Lymphoblastic Leukemia (B-ALL).
A multi-institutional study found that 1 in 6 youths fill an opioid prescription prior to surgery, and 3% of patients were still filling opioid prescriptions three to six months after surgery, indicating persistent opioid use and possible opioid dependence.
A new study from researchers found that LGBTQ+ youth were more likely to experience depression and thoughts and attempts of suicide than non-LGBTQ+ youth, yet the prevalence of these mental health symptoms were significantly reduced when LGBTQ+ youths reported support from their parents.
Researchers found that young children between the ages of 5 and 12 were more likely to experience a concussion from recreation and other non-sport activities, yet those injuries were not seen by specialists until days later compared with sports-related concussions in the same age group.
While doctors understand that adolescents have certain unique health questions and concerns, several challenges prevent this critical information from being collected in a systematic way and providing appropriate referrals across a wide group of patients.
Researchers developed, implemented and successfully tested a nutrition screener to improve access to healthy resources for families eligible for federally funded food benefits.
Researchers have identified a causal genetic variant strongly associated with childhood obesity. The study provides new insight into the importance of the hypothalamus of the brain and its role in common childhood obesity and the target gene may serve as a druggable target for future therapeutic interventions.
An international consortium published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).
As patients with congenital heart diseases live longer, researchers are attempting to understand some of the other complications they may face as they age. In a new study, a team from Children鈥檚 Hospital of Philadelphia (CHOP) used state-of-the-art technologies to understand the underlying biology of Fontan-associated liver disease (FALD).
In the wake of the COVID-19 pandemic, many families worried about the long-term effects posed by the SARS-COV-2 virus. Now, researchers found that a SARS-COV-2 infection likely does not increase the risk of asthma development in pediatric patients. The findings were published today in the journal Pediatrics.
CAR T cell therapy has revolutionized the way certain types of cancer are treated, and the longer those CAR T cells live in a patient鈥檚 body, the more effectively they respond to cancer. Now, researchers have found that a protein called FOXO1 improves the survival and function of CAR T cells, which may lead to more effective CAR T cell therapies and could potentially expand its use in difficult-to-treat cancers.
Researchers found that teens and young adults with mood disorders, such as depression and bipolar disorder, were 30% less likely to obtain their driver鈥檚 license than peers without such disorders. Additionally, those youths with mood disorders experienced a slightly elevated risk of crashing.
In the first clinical trial of a targeted pharmacologic therapeutic for mild traumatic brain injury in pediatric patients, scientists have found preliminary evidence that adolescents and young adults with concussion who take a specific formulation of branched chain amino acid (BCAA) supplements after injury experience faster symptom reduction and return to physical activity.
Researchers identified a key metabolite in cells that helps direct immune responses and explains at a single cell level why immune cells that most efficiently recognize pathogens, vaccines, or diseased cells grow and divide faster than other cells.
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