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Editas Medicine Inc. (NASDAQ:EDIT), and Allergan plc (NYSE:AGN) aims to dose first LCA10 patients for CRISPR gene therapy in Q1 2020

Editas Medicine, Inc. (NASDAQ: EDIT), based in Dublin, Ireland and Cambridge, Massachusetts, and Allergan plc (NYSE: AGN), also headquartered in Dublin, Ireland have announced that the first potential patient has been successfully screened to administer EDIT-101 by early 2020.  “EDIT-101” is a CRISPR-based technology for the treatment of Leber Congenital Amaurosis type 10 (LCA10).  CRISPR […]

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Pentosan polysulfate sodium (PPS), a treatment for interstitial cystitis, has raised concerns for a contraindication of macular disease on long term effects.

A clinical research report in the British Journal of Ophthalmology has indicated that the use of pentosan polysulfate sodium (PPS, registered as “Elmiron”, Janssen Pharmaceuticals) is now associated with a new diagnosis of macular disease at a seven-year follow-up in a large retrospective matched cohort study.  PPR / Elmiron is marketed for the treatment of […]

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A research study of Stargardt disease (STGD1) highlights inter-sibling variability between genotype-phenotype

Research at the Radboud University Medical Centre in Nijmegen, the Netherlands, has reported a phenotypic discordance between siblings with STGD1 having the same ABCA4 variants.  While fundus autofluorescence images appear to compare phenotypic outcomes between siblings, the study identifies substantially different functional outcomes. The research study may throw up significant challenges for clinical management and […]

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