Researchers Use Gene Editing with CRISPR to Treat Lethal Lung Diseases before Birth

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...
14-Apr-2019 7:00 PM EDT Add to Favorites

Gene-Based Factor VIIa Prevents Bleeding Episodes in Animals with Hemophilia

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...
15-Apr-2019 2:30 PM EDT Add to Favorites

Proton Therapy Demonstrates Efficacy and Reduced Toxicity in Largest-Ever Cohort of Pediatric High-Risk Neuroblastoma Patients

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...
10-Apr-2019 9:45 AM EDT Add to Favorites

Children Requiring Thyroid Surgery Experience Better Outcomes At High-Volume Surgery Centers

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...
4-Apr-2019 12:05 PM EDT Add to Favorites

Pediatric Cell Atlas Will Map Single-Cell Changes for a Deeper View of Child Health and Disease

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...
21-Mar-2019 10:15 AM EDT Add to Favorites

In Comparison of Standard Treatment to Sustained Inflations for Extremely Premature Infants, Standard Treatment Prevails

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...
26-Mar-2019 4:45 PM EDT Add to Favorites

New Computational Tool Harnesses Big Data and Deep Learning to Illuminate 'Dark Matter' of the Transciptome

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.
25-Mar-2019 5:00 PM EDT Add to Favorites

Patients Bear Increased Financial Burden for Growth Hormone Treatment Despite FDA Approval and Lower Total Cost

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.
21-Mar-2019 11:05 AM EDT Add to Favorites

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