Base Editing of Gamma-Globin Promoters Increases Fetal Hemoglobin in Sickle Cell Disease
Interim phase 1-2 data show risto-cel achieves 60% fetal hemoglobin and eliminates severe vaso-occlusive crises in 31 patients.
Black Children Show High Skin Disease Burden but Low Dermatology Use
A multicenter study of 536,776 pediatric patients reveals significant racial gaps in specialty care access for common skin conditions.
Six-Item Symptom Tool and Vestibular Screen Improve Concussion Diagnosis Accuracy
A streamlined 6-item index combined with a brief vestibular-ocular screen shows higher specificity than the standard 22-item checklist.
ECPR May Improve Survival in Pediatric Out-of-Hospital Cardiac Arrest
A multicenter registry study finds higher survival and neurologic recovery rates with extracorporeal support despite wide confidence intervals.
Early Language and Motor Skills Predict Adolescent Independence in Autism
A decade-long longitudinal study identifies preschool markers that determine functional daily living outcomes for autistic teenagers.
Water Access and Vaccination Linked to Lower Pediatric HIV Mortality
Meta-regression of 67 cohorts identifies water access, pneumococcal vaccination, and maternal education as key predictors of survival.
Active Support Increases Injury Prevention Program Use in Female Athletes
In-person workshops for coaches tripled the use of injury prevention protocols, though direct impact on ACL and concussion rates remains unclear.
US Vitiligo Patients Experience Brief Treatment Durations and High Untreated Rates
A retrospective cohort study of 24,949 patients reveals significant gaps in long-term management and frequent use of short-term therapies.
Abrocitinib Shows Faster Itch Relief Than Dupilumab in Atopic Dermatitis
A post hoc analysis of JADE trials finds oral JAK1 inhibition reaches an itch-free state sooner, correlating with better sleep and quality of life.
Fetal MRI Scoring System Quantifies Severity in Trisomy 13, 18, and CHARGE
A new morphological score and network analysis map phenotypic heterogeneity to assist in prenatal counseling and management planning.
Triplet Immunotherapy Shows Durable Response in Relapsed Hodgkin Lymphoma
Adding ipilimumab to nivolumab and brentuximab vedotin may improve long-term disease control for patients deferring stem cell transplant.
Biological Maturation Predicts Sex-Specific Injury Risk in Junior Tennis Athletes
Boys face the highest injury burden during their peak growth spurt, while girls are most vulnerable 18 months after reaching maturation.