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Choroideremia clinical trials at UC Health
2 research studies open to new patients

  • Efficacy and Safety of AAV2-REP1 for the Treatment of Choroideremia

    open to eligible males ages 18 years and up

    The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of AAV2-REP1 in subjects with Choroideremia.

    at UCLA

  • Natural History of the Progression of Choroideremia Study

    open to eligible males ages 18 years and up

    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.

    at UCLA

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