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ABSTRACT: Importance
Sensitive outcome measures for disease progression are needed for treatment trials of Stargardt disease.Objective
To estimate the progression rate of atrophic lesions in the prospective Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) study over a 12-month period.Design, setting, and participants
This multicenter prospective cohort study was conducted in an international selection of tertiary referral centers from October 21, 2013, to February 15, 2017. Patients who were affected by Stargardt disease, aged 6 years and older at baseline, and harboring disease-causing variants of the ABCA4 gene were enrolled at 9 centers in the United States, United Kingdom, and continental Europe. Data analysis occurred from November
SUBMITTER: Strauss RW
PROVIDER: S-EPMC6681653 | biostudies-literature | 2019 Oct
REPOSITORIES: biostudies-literature