Niemann-Pick type C as a cause of progressive intellectual and neurological deterioration in childhood.

Aim: To describe the cases of Niemann-Pick type C (NP-C) disease in a United Kingdom epidemiological study of progressive intellectual and neurological deterioration in childhood.

Method: Paediatricians notified cases via the British Paediatric Surveillance Unit between 1997 and 2015.

Results: Fifty-three NP-C patients were identified: 29 females, 24 males.

Leukodystrophies and genetic leukoencephalopathies in childhood: a national epidemiological study.

Aim: To report on the epidemiology of the brain white matter disorders of children identified via a national prospective study.

Method: Since 1997 a study of UK children with progressive intellectual and neurological deterioration (PIND) has used the British Paediatric Surveillance Unit system to identify children with progressive neurodegenerative disease. This paper reports on children in the study with brain white matter disorders.

Clinical features of narcolepsy in children vaccinated with AS03 adjuvanted pandemic A/H1N1 2009 influenza vaccine in England.

Aim: The aim of this study was to investigate whether children in England with narcolepsy who received the ASO3 adjuvanted pandemic A/H1N1 2009 influenza vaccine (Pandemrix) differed clinically from unvaccinated patients.

Method: A retrospective review was conducted in children with narcolepsy diagnosed by sleep centres and paediatric neurologists in 16 English hospitals. The inclusion criteria were patient age 4 to 18 years, onset of narcolepsy after January 2008, and diagnosis by the time of the key data-gathering visit in 2011.

Pandemic A/H1N1 2009 influenza vaccination, preceding infections and clinical findings in UK children with Guillain-Barré syndrome.

Objective: To record clinical findings in all new cases of Guillain-Barré syndrome (GBS) or Fisher syndrome (FS) in UK children in the 2 years following September 2009 and determine the proportion temporally associated with recent infections, pandemic H1N1 (2009) strain influenza vaccination or seasonal influenza vaccination.

Design: A prospective UK-wide epidemiological study using the British Paediatric Surveillance Unit system.

Patients: Children aged 16 years or less meeting the Brighton Collaboration criteria for GBS or FS.

Risk of narcolepsy in children and young people receiving AS03 adjuvanted pandemic A/H1N1 2009 influenza vaccine: retrospective analysis.

Objective: To evaluate the risk of narcolepsy in children and adolescents in England targeted for vaccination with ASO3 adjuvanted pandemic A/H1N1 2009 vaccine (Pandemrix) from October 2009.

Design: Retrospective analysis. Clinical information and results of sleep tests were extracted from hospital notes between August 2011 and February 2012 and reviewed by an expert panel to confirm the diagnosis.