ProQR eyes its first big test

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via Despite being an ultra-rare disease, Leber's congenital amaurosis type 10 (LCA10), a form of inherited blindness, has attracted both Proqr Therapeutics and Editas Medicine. The latter stumbled last year, and Proqr will hope to do better with its lead project, sepofarsen, on which pivotal data are due this quarter.

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