SMA is a motor neuron disease characterised by generalised muscle atrophy and weakness. It is caused by the dysfunction and eventual death of motor neurons, due to deletions of SMN1 in 95% of patients ...

Objective To investigate a possible association between fever admissions and 4 component Meningococcal B (4CMenB). Design 4CMenB is given at 8 and 16 weeks in the first year of life. Self-controlled ...

Background Over the past 2–3 years at the Southwest Peninsula tertiary neonatal unit in Plymouth, the authors have observed an increase in the number of clinically well term infants being screened and ...

Objective Hypertrophic cardiomyopathy (HCM) is an important predictor of long-term outcomes in Friedreich’s ataxia (FA), but the clinical spectrum and survival in childhood is poorly described. This ...

Cystic fibrosis is a disease that still causes a reduced life expectancy. The treatment burden remains high for affected individuals with often a combination of multiple oral and inhaled medications, ...

Introduction Bronchiolitis is a very common acute respiratory illness affecting infants and is a significant cause of acute morbidity in the first 12 months of life. The diagnosis of bronchiolitis is ...

Objectives Before 2003, most children with acute lymphoblastic leukaemia (ALL) abandoned treatment, with only approximately 30% treated in China. With the development of national insurance for ...

Objective methods are being used increasingly for the quantification of the amount of physical activity, intensity of physical activity and amount of sedentary behaviour in children. The accelerometer ...

Objectives To provide up-to-date information on the use of long-term ventilation (LTV) in the UK paediatric population and to compare the results with data collected 10 and 20 years previously. Design ...

We encounter, occasionally, parents who are incapable of making medical decisions on behalf of their incompetent or incapacitated children. When the incapable parent is themselves a child; measures ...

Objectives Develop a score summarising how successfully a child with any surgical condition has been treated, and test the clinical validity of the score. Design Discrete choice experiment (DCE), and ...

Kawasaki disease (KD) is an acute self-limiting inflammatory disorder, associated with vasculitis, affecting predominantly medium-sized arteries, particularly the coronary arteries. In developed ...