Last updated on August 2018

Natural History of the Progression of Choroideremia Study

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Brief description of study

Choroideremia (CHM) is a rare, X-linked recessive, degenerative disease of the retina which begins in childhood with loss of night vision and gradually progresses to legal blindness by the fifth decade. There are no approved treatements for CHM and the disease is poorly characterised given its rare nature and scarscity of available data. The objective of this study is to collect natural history data from a large cohort of CHM patients in order to evaluate possible efficacy measures that could be utilised in future interventional trials of novel therapies.