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The Progression of the Stargardt Disease Type 4 (ProgStar-4) Study: Design and Baseline Characteristics (ProgStar-4 Report No. 1) by Rupert W. Strauss; Beatriz Muñoz; Mohamed I. Ahmed; Millena Bittencourt; Etienne M. Schönbach; Michel Michaelides; David Birch; Eberhart Zrenner; Ann-Margret Ervin; Peter Charbel Issa; Jun Kong; Yulia Wolfson; Mahmood Shah; Saghar Bagheri; Sheila West; Hendrik P.N. Scholl; for the ProgStar-4 Study Group is a Medicine article available to read on EtoBox.

What is The Progression of the Stargardt Disease Type 4 (ProgStar-4) Study: Design and Baseline Characteristics (ProgStar-4 Report No. 1) about?

**__Background/Aims:__** To describe the design and baseline characteristics of patients enrolled in the multicenter, prospective natural history study of Stargardt disease type 4. **__Methods:__** Fifteen eligible patients aged 6 years and older at baseline, harboring disease-causing variants in the __PROM1__ gene, and with specified ocular lesions were enrolled. They were examined at baseline using a standard protocol, with 6 monthly follow-up visits for a 2-year period including best-corrected ETDRS visual acuity, spectral-domain optical coherence tomography, fundus autofluorescence (FAF), mesopic and scotopic microperimetry (MP). Areas of definitely decreased FAF (DDAF) and questionably decreased FAF were outlined and quantified on FAF images. **__Results:__** Amongst the 15 patients (29 eyes) that were enrolled at 5 centers in the USA and Europe, 10 eyes (34.5%) had areas of DDAF with an average lesion area of 3.2 ± 3.5 mm^2^ (range 0.36–10.39 mm^2^) at baseline. The mean retinal sensitivity of the posterior pole derived from mesopic MP was 8.8 ± 5.8 dB. **__Conclusions:__** Data on disease progression in __PROM1__-related retinopathy from this study will contribute to the cha

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Author

Rupert W. Strauss; Beatriz Muñoz; Mohamed I. Ahmed; Millena Bittencourt; Etienne M. Schönbach; Michel Michaelides; David Birch; Eberhart Zrenner; Ann-Margret Ervin; Peter Charbel Issa; Jun Kong; Yulia Wolfson; Mahmood Shah; Saghar Bagheri; Sheila West; Hendrik P.N. Scholl; for the ProgStar-4 Study Group

Publisher

S. Karger AG

Published

2018

Language

EN

Field

Medicine (Health Sciences)