In the United States, there are currently more adults living with cerebral palsy than children. Despite this, the Centers for Disease Control and Prevention still label cerebral palsy as “the most common motor disability in childhood.”
Researchers at Children’s 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.
Recent advances in bladder cancer treatments may offer hope of curative care to more patients, including those with high-risk localized, muscle-invasive disease, according to a New England Journal of Medicine editorial published by Matthew Milowsky, MD, FASCO, a bladder cancer expert at UNC School of Medicine and UNC Lineberger Comprehensive Cancer Center.
The first powered randomized trial examining early intervention with transcatheter aortic valve replacement (TAVR) in patients with asymptomatic, severe aortic stenosis (AS) found this strategy to be both a safe and effective alternative to clinical surveillance (CS).
Findings were reported today at TCT 2024, the annual scientific symposium of the Cardiovascular Research Foundation (CRF). TCT is the world’s premier educational meeting specializing in interventional cardiovascular medicine. Results were also published simultaneously in the New England Journal of Medicine.
A syndrome called cachexia, which triggers unexplained loss of weight and muscle mass, causes severe illness and death among patients with cancer and other serious health conditions.
A review of what's known about the crisis in physician well-being, and what can be done to address it, also suggests a need for action by health systems, and more research.
University of California San Diego School of Medicine researchers find advanced AI could lead to easier, faster and more efficient hospital quality reporting.
Updated results from a phase 3 clinical trial are expected to change the way advanced stage classic Hodgkin lymphoma (cHL) is treated. Details appear in a study published today in The New England Journal of Medicine. Drs. Kara Kelly of Roswell Park Comprehensive Cancer Center and Sharon Castellino of Children’s Healthcare of Atlanta and Emory University School of Medicine/Winship Cancer Institute of Emory University served as pediatric leads of this important trial in children and adults with lymphoma.
According to findings from a multicenter study led by Johns Hopkins Medicine and released today in The New England Journal of Medicine (NEJM), transplanting kidneys from deceased donors who had the human immunodeficiency virus (HIV) to recipients with HIV is safe. Perhaps more importantly, the study authors also found that HIV-to-HIV kidney transplants are comparable in effectiveness to those using organs from donors without HIV.
A treatment that rallies the immune system to destroy cancer raised the survival rate for advanced Hodgkin lymphoma patients to a remarkable 92 percent, suggesting a new standard therapy for the disease. The New England Journal of Medicine published the innovative clinical trial results this week.
A new systematic review led by researchers in Hamilton, Canada has shown that specialized in-bed cycling therapy, when used in the intensive care unit with critically ill patients, leads to better physical function and a one-day shorter length of stay in the ICU.
An international placebo-controlled study led by Cedars-Sinai suggests that a targeted drug therapy that was developed by researchers at Cedars-Sinai is safe and effective at helping people with moderate to severe ulcerative colitis reach clinical remission.
CLEVELAND: A clinical trial has demonstrated that the cancer drug pomalidomide is safe and effective in treating hereditary hemorrhagic telangiectasia (HHT), a rare bleeding disorder that impacts more than 1 in 5,000 people worldwide.
Avian influenza A(H5N1) virus, which spread to cattle and infected 14 people this year, was detected using virome sequencing in the wastewater of 10 Texas cities by researchers at UTHealth Houston and Baylor College of Medicine.
A brain-computer interface developed by UC Davis Health accurately translates brain signals into speech. The device implanted in the brain of a man with ALS is the most accurate system of its kind.
A new drug for treatment of a type of brain cancer, called IDH-mutant low-grade glioma, was approved Aug. 6 by the U.S. Food and Drug Administration (FDA). The promising new drug stems from a 2008 genetic discovery made at the Johns Hopkins Kimmel Cancer Center.
When it comes to caring for children with congenital adrenal hyperplasia (CAH), avoiding the side effects of traditional treatments has long been a challenge. But in a recent phase 3 clinical trial, a new drug called crinecerfont showed safety and efficacy in children with classic CAH, the severe form of this condition.
En un nuevo estudio multicéntrico internacional dirigido por el Centro Oncológico Integral de Mayo Clinic, investigadores encontraron que las personas con el subtipo de leucemia linfoblástica aguda precursores de células B (LLA-BCP), que tampoco presentaban una anomalía genética conocida como cromosoma Filadelfia y que estaban en remisión y sin rastros de cáncer, mostraron tasas de supervivencia significativamente más altas cuando se agregó blinatumomab al tratamiento de quimioterapia.