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Structural determinants of long term functional outcomes in young children with cystic fibrosis

Structural determinants of long term functional outcomes in young children with cystic fibrosis BACKGROUND: Accelerated lung function decline in

Primary Nasal Epithelial Cells as a Surrogate Cell Culture Model for Type-II Alveolar Cells to Study ABCA-3 Deficiency

ATP Binding Cassette Subfamily A Member 3 (ABCA-3) is a lipid transporter protein highly expressed in type-II alveolar (AT-II) cells. Mutations in ABCA3 can result in severe respiratory disease in infants and children. To study ABCA-3 deficiency in vitro, primary AT-II cells would be the cell culture of choice although sample accessibility is limited. Our aim was to investigate the suitability of primary nasal epithelial cells, as a surrogate culture model for AT-II cells, to study ABCA-3 deficiency.

Parental occupational paint exposure and risk of childhood leukemia in the offspring: findings from the Childhood Leukemia International Consortium

Using individual data from fathers of 8,185 cases and 14,210 controls, the pooled OR for paternal exposure around conception and risk of acute lymphoblastic...

Parental occupational pesticide exposure and the risk of childhood leukemia in the offspring: Findings from the childhood leukemia international consortium

Maternal occupational pesticide exposure during pregnancy and/or paternal occupational pesticide exposure around conception have been suggested to increase...

Early rheumatic heart disease is a recognized intermediate on the pathway to advanced rheumatic heart disease

Rheumatic heart disease is a major cause of premature cardiovascular morbidity and mortality globally. Over the past decade, echocardiographic screening has changed our understanding of the natural history of RHD, revealing a high burden of clinically silent, mild RHD among people who cannot recall a history of preceding acute rheumatic fever. This viewpoint outlines the evidence that this earliest form of rheumatic heart disease, only detectable through echocardiographic screening, is an intermediate stage that many, but not all, individuals may pass through on the pathway to advanced rheumatic heart disease.

BEAT-CF (Bayesian Evidence Adaptive Treatment for people with Cystic Fibrosis): description of a prospective cohort for nested studies in cystic fibrosis

Despite recent improvements in treatment modalities for cystic fibrosis (CF), there is currently limited evidence and a lack of consensus regarding optimal treatment strategies for the different aspects of CF, including pulmonary exacerbations (PEx). We aimed to establish a prospective cohort of people with CF (pwCF) to evaluate alternative approaches to managing CF in the era of modulator therapies.

Treat early to protect lungs

Early intervention is being touted as the key to preventing lung damage in children with cystic fibrosis.

An introduction to clinical trial design

This manuscript will give a brief overview of clinical trial design including the strengths and limitations of various approaches

Research Note: Adaptive trials

This Research Note has explored the strengths, risks and potential complexity of adaptive trials

Thomas Iosifidis

Research Officer