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The COVID-19 pandemic is one of the most serious global challenges to delivering affordable and equitable treatment to children with cancer we have witnessed in the last few decades. This Special Report aims to summarize general principles for…
BACKGROUND: Mucopolysaccharidosis type I (MPS I) is a rare lysosomal storage disease subdivided into three phenotypes of increasing severity: Scheie, Hurler-Scheie and Hurler. To gauge the effectiveness of treatments and to determine the load likely…
OBJECTIVES: The goals were (1) to describe and to characterize pediatric clinical research networks (PCRNs) in the United States and Canada, (2) to identify PCRN strengths and weaknesses, (3) to evaluate the potential for collaboration among PCRNs,…
Many children in the United States fail to reach their full health and developmental potential. Disparities in their health and well-being result from the complex interplay of multiple social and environmental determinants that are not adequately…
Undernutrition, growth failure, overweight, micronutrient deficiencies, and osteopenia are nutritional comorbidities that affect the neurologically impaired child. Monitoring neurologically impaired children for nutritional comorbidities is an…
New regulatory initiatives have been designed to ensure that new drugs and biologicals include adequate pediatric labeling for the claimed indications at the time of, or soon after, approval. However, because such labeling may not immediately be…
There is an urgent need for robust empirical data to guide the assessment and treatment of patients near the end of life. Because they are important providers of end-of-life care in this country, hospices have an important role to play in…