Results 81 to 90 of about 2,171,498 (340)
ABSTRACT Objective Prognostication of disease course and prediction of treatment response in multiple sclerosis is an unmet need. We compared the performance of serum neurofilament light chain Z scores (age‐ and BMI‐adjusted) with absolute concentrations for the prediction of response to disease‐modifying therapy.
Maximilian Einsiedler+43 more
wiley +1 more source
Introduction Pain in pediatric oncology patients is often undertreated due to a lack of timely assessment and inefficient communication between health care workers. Improper pain assessment is a leading cause of poorly managed pain in children.
Bashir Ahmed Khan+3 more
doaj +1 more source
The First Five Minutes: Enhancing Simulation Education for First-Year Pediatric Residents [PDF]
We are looking at the feasibility of redesigning the existing simulation education for first-year residents within the Children’s Hospital of Richmond at VCU’s pediatric residency program to increase learning opportunities and to enhance exposure to ...
Coe, Megan E.
core +1 more source
Gene Therapy for Pediatric Cancer: State of the Art and Future Perspectives [PDF]
While modern treatments have led to a dramatic improvement in survival for pediatric malignancy, toxicities are high and a significant proportion of patients remain resistant.
Biagi, Ettore+3 more
core +5 more sources
ABSTRACT Objective Neurodegeneration with brain iron accumulation (NBIA) comprises rare genetic disorders characterized by predominantly extrapyramidal symptoms and iron deposition in the basal ganglia. Conventional magnetic resonance imaging (MRI) detects qualitative changes but cannot accurately quantify iron accumulation. Quantitative susceptibility
Özge Uygun+19 more
wiley +1 more source
IntroductionLipodystrophy syndromes comprise a group of rare endocrine disorders characterized by the generalized or partial loss of adipose tissue.
Rebecca J. Brown+12 more
doaj +1 more source
Overcoming steroid resistance in T cell acute lymphoblastic leukemia [PDF]
In a Perspective, Pieter Van Vlierberghe and Steven Goossens discuss Meijerink and colleagues' findings on steroid resistance in pediatric T cell acute lymphoblastic ...
Goossens, Steven+1 more
core +3 more sources
Long‐Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons
ABSTRACT Objectives This ongoing, open‐label extension study is evaluating the long‐term safety, tolerability, and efficacy of givinostat, a Class I and II histone deacetylase inhibitor, in patients with Duchenne muscular dystrophy (DMD). Methods The recruited patients completed one of two prior clinical studies (one Phase 2 and one Phase 3 [EPIDYS ...
Craig M. McDonald+74 more
wiley +1 more source
Practice Recommendations for Genetic Testing of Ataxias
ABSTRACT Objective Over the past decade, significant advances in genetic testing for ataxia have improved diagnostic accuracy, informed clinical trial eligibility, guided treatment decisions, and enabled cascade testing of at‐risk relatives. While guidance exists for other neurogenetic conditions, there are no standardized guidelines on genetic ...
Sharan R. Srinivasan+7 more
wiley +1 more source