Results 11 to 20 of about 86,084 (265)
Orphanet Journal of Rare Diseases, 2020 Background The Office of Orphan Products Development (OOPD) of the United States (U.S.) Food and Drug Administration (FDA) has awarded over 700 grants to conduct clinical trials of medicals products for rare diseases since 1983, leading to over 70 ...
Kathleen L. Miller +4 more
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Joint effort to target the orphan of the orphan [PDF]
Blood, 2019 In this issue of Blood, Cuvelier and colleagues from the Applied Biomarkers of Late Effects of Childhood Cancer (ABLE) Consortium evaluate, for the first time, the performance of the National Institutes of Health Consensus Criteria (NIH-CC) for diagnosing chronic graft-versus-host disease (cGVHD) in pediatric patients in a prospective multicenter trial.
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Journal of Virus Eradication, 2016 An analysis of the impact of orphanhood at antiretroviral therapy (ART) initiation on HIV outcomes in Asia included 4300 children; 51% were male. At ART initiation, 1805 (42%) were non-orphans (median age: 3 years), 1437 (33%) were single orphans (6 ...
B.u.i.V.u. Huy +21 more
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Transport study of interleukin-1 inhibitors using a human in vitro model of the blood-brain barrier
Brain, Behavior, & Immunity - Health, 2021 The proinflammatory cytokine Interleukin-1 (IL-1), with its two isoforms α and β, has important roles in multiple pathogenic processes in the central nervous system.
Elisabet O. Sjöström +6 more
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Proceedings of the National Academy of Sciences, 1997 The recent discovery of several new endogenous opioid peptides has generated excitement over their potential physiological roles. For example, endomorphins are likely to have well defined functions due to their affinity and selectivity for a class of extensively studied opioid receptors (1).
J R, Walker, G F, Koob
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Census of solo LuxR genes in prokaryotic genomes
Frontiers in Cellular and Infection Microbiology, 2015 luxR genes encode transcriptional regulators that control acyl homoserine lactone-based quorum sensing (AHL QS) in Gram negative bacteria. On the bacterial chromosome, luxR genes are usually found next or near to a luxI gene encoding the AHL signal ...
Sanjarbek eHudaiberdiev +7 more
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An insight into bronchiectasis: Causes, clinical features, and treatment practices
Indian Journal of Respiratory Care, 2020 Introduction: Bronchiectasis is a common, progressive respiratory disease characterized by irreversibly dilated, damaged, and thickened bronchi. It is present as a clinical syndrome of chronic cough, sputum production, and recurrent lower respiratory ...
Laxmi Devi +4 more
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Mobile DNA, 2021 Background Retrotransposons have been implicated as causes of Mendelian disease, but their role in autism spectrum disorder (ASD) has not been systematically defined, because they are only called with adequate sensitivity from whole genome sequencing ...
Rebeca Borges-Monroy +9 more
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Classic galactosemia: features of diagnosis and treatment
Zdorovʹe Rebenka, 2018 Background. Galactosemia type I, or classic galactosemia (CG), is the most severe form of hereditary disorders of carbohydrate metabolism, in particular, galactose. Galactosemia is not included in the program of massive neonatal screening in Ukraine. The
N.O. Pichkur +2 more
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