Flagship Journal

The rare diseases Duchenne and Becker are X-linked recessive neuromuscular disorders.1 They are characterised by progressive…

Climate change is happening, with the evidence clearly demonstrating an increase in global temperatures; extreme weather events…

Nebulisers are devices used to rapidly deliver high doses of medication to the lungs in people with respiratory disease…

One in 10 people may be affected by a rare disease (RD) in their lifetime.1 This is the surprising paradox of RDs. While each disease…

Children diagnosed with congenital heart disease (CHD) have an increased risk of developing neurodevelopmental disabilities…

Zinc salts are one of three approved treatments for the autosomal recessive condition Wilson’s disease (WD). Their use was first described…

A previously healthy 17-year-old male was transferred from a community hospital to the authors’ tertiary paediatric emergency department…

Dermatitis artefacta (DA), also known as artefactual skin disease, is a rare psychocutaneous disorder characterised by…