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Inherited retinal diseases (IRDs), known as single-gene disorders impacting the retina, are highly hard to diagnose as they are unusual and involve alterations in one of several candidate genes.
The FDA fast-tracked the approval on the basis of early trial results.
Among them is a two-year-old girl who was diagnosed with a disease that causes severe developmental disorder.
/PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines...
Enrollment progressing in Phase 1b Multiple-Ascending Dose (MAD) study of RGLS8429 in patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD) Top-line data in the first cohort of RGLS8429-treated ADPKD patients anticipated in second half of ...