Institute of Immunity and Transplantation



In observational trials investigators observe the subject and measure outcomes without intervening or actively managing the study.

Patients are observed to establish the impact of disease and of existing treatments on the physical/psychological wellbeing of the patient.

Careful examination and follow up of patients having standard therapies improves our understanding of treatment and disease.


Lysosomal storage disorders are rare conditions which vary between each condition and between patients with the same condition.

Careful documentation and annotation of the clinical pictures in clinical trials and registries improves our understanding of the natural history, treatment need and likely prognosis.

The DETECT Study compared multiple, non-invasive screening tools and their combinations with right heart catheterisation, the gold standard diagnostic test for pulmonery arterial hypertension (PAH). The 8 item DETECT score has provided a new way of assessing risk of PAH in scleroderma, allowing patients at high risk to be identified for right heart cathetherisation.

With standard treatment and more organised care in environments like the Institute, survival of the worst forms of many diseases have significantly improved over a decade.