A phase 1 clinical study shows that gene therapy extends survival of patients and supports achievement of milestones previously unseen in the natural course of the disease. A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1), according to a study published in the New England Journal of Medicine.
Adolescents with severe obesity who had bariatric surgery showed significant improvements in cardiovascular disease risk factors, according to the most recent “Teen Longitudinal Assessment of Bariatric Surgery” (Teen-LABS) study, published online today by Pediatrics.
A new study led by the Center for Injury Research and Policy at Nationwide Children’s Hospital used data from a national survey to examine the effectiveness of state-level cellphone laws in decreasing teens’ use of cellphones while driving.
Acute lymphoblastic leukemia (ALL), a malignancy of the white blood cells, is a common childhood cancer. Understanding the genomic changes underlying ALL has led to the development of new, targeted therapies, especially for patients with high-risk subtypes.
A Child's Voice in the World
Increasing access to care for our most vulnerable children can change the world.
There are too few resources for children who need them, and the stigma attached to behavioral health issues keeps some families from seeking out the help they need.
Over the past five decades, the health of America’s children has improved considerably. Unfortunately, those improvements were not evenly distributed. Most American children are healthy and have promising futures. Children in poverty, on the other hand, infrequently move up the social ladder in the United States and are subject to higher rates of hunger, homelessness, violence and other challenges.
Adult emergency departments are overwhelmed with mental health visits, and the problem worsens every year. There are many explanations, but the most basic is that we are missing opportunities to intervene before patients have mental health crises. Many missed opportunities actually come in childhood.
Officials, Republican and Democrat, really do want the best for their constituents. Maybe we’re optimists in the health care field, but our experience has convinced us that it’s true. So much of the health care conversation though, is conducted by people who don’t have front-line experience helping patients. We do. It’s up to us, as members of the medical community, to stand up for those who need our care.