Progression of Stargardt disease as determined by fundus autofluorescence in the retrospective progression of Stargardt disease study (ProgStar report No. 9)
RW Strauss, B Muñoz, A Ho, A Jha… - JAMA …, 2017 - jamanetwork.com
Importance Sensitive outcome measures for disease progression are needed for treatment
trials of Stargardt disease. Objective To describe the yearly progression rate of atrophic
lesions in the retrospective Progression of Stargardt Disease study. Design, Setting, and
Participants A multicenter retrospective cohort study was conducted at tertiary referral
centers in the United States and Europe. A total of 251 patients aged 6 years or older at
baseline, harboring disease-causing variants inABCA4 (OMIM601691), enrolled in the study …
trials of Stargardt disease. Objective To describe the yearly progression rate of atrophic
lesions in the retrospective Progression of Stargardt Disease study. Design, Setting, and
Participants A multicenter retrospective cohort study was conducted at tertiary referral
centers in the United States and Europe. A total of 251 patients aged 6 years or older at
baseline, harboring disease-causing variants inABCA4 (OMIM601691), enrolled in the study …