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Highly sensitive measurements of disease progression in rare disorders: Developing and validating a multimodal model of retinal degeneration in Stargardt disease

BACKGROUND: Each inherited retinal disorder is rare, but together, they affect millions of people worldwide. No treatment is currently available for these blinding diseases, but promising new options—including gene therapy—are emerging. Arguably, the most prevalent retinal dystrophy is Stargardt dis...

Ausführliche Beschreibung

Bibliographische Detailangaben
Hauptverfasser: Lambertus, Stanley, Bax, Nathalie M., Fakin, Ana, Groenewoud, Joannes M. M., Klevering, B. Jeroen, Moore, Anthony T., Michaelides, Michel, Webster, Andrew R., van der Wilt, Gert Jan, Hoyng, Carel B.
Format: Online Artikel Text
Sprache:English
Veröffentlicht: Public Library of Science 2017
Schlagworte:
Online Zugang:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5371327/
https://www.ncbi.nlm.nih.gov/pubmed/28355279
http://dx.doi.org/10.1371/journal.pone.0174020