Rare disease

Basic page

BPSU study - Behçet’s syndrome

Surveillance of Behçet’s syndrome in children and young people concluded in May 2017. The study team hope to ascertain the incidence and prevalence of Behçet’s syndrome in children and young people. They also hope to examine the clinical presentation and management of the syndrome. A paper has n...
Basic page

BPSU study - Pierre Robin Sequence

Surveillance of Pierre Robin Sequence in the UK and ROI concluded in January 2017. The study team is hoping to identify the birth prevalence of Pierre Robin Sequence, outline its clinical presentation, initial management and 12 month clinical outcomes. The study is being led by Dr Marie Wright, winn...
Basic page

BPSU study - Nutritional rickets

Surveillance of nutritional rickets presenting to secondary care in children and young people concluded in March 2017. The study team hoped to ascertain the incidence of nutritional rickets and presentational features of the condition. The study group has now published its findings in the Archiv...

Great Ormond Street Hospital Charity & LifeArc - Translational Research Accelerator Grants [OPEN]

Through this scheme, GOSH Charity and LifeArc are inviting milestone-driven applications that aim to improve the prevention, diagnosis, prognosis, or treatment of rare disease or that focus on developing research tools that increase the efficiency of developing interventions in rare disease. For the first time, the 2025 call will be open nationally to academic researchers from eligible Higher Education Institutions (HEIs) across the United Kingdom.
Award programme/theme
Project grant
Funding amount range
£100,001 to £250,000
Maximum duration
3 years
Submission deadline