• FDA clears IND application for ProQR's Usher syndrome treatment

    6 days ago - By Healio

    The FDA has cleared an investigational new drug application for QR-421a for Usher syndrome type 2, ProQR Therapeutics announced in a press release.
    QR-421a, an investigational RNA-based oligonucleotide, addresses the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene, the release said.
    The company plans to begin the phase 1/2 STELLAR clinical trial of QR-421a in early 2019. The first-in-human, double-masked, international, randomized study will include approximately 18 adults, with preliminary data
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  • Top stories in endocrinology: FDA suspends trial involving investigational diabetes drug, issues guidance for blood glucose meters

    6 days ago - By Healio

    Among the most-read stories in endocrinology this week was the announcement that the FDA suspended the trial of an investigational drug for treating type 2 diabetes and that the federal agency had issued two draft guidances for blood glucose meters.
    Other popular stories included a position statement from two endocrinology societies that identified multiple stages of type 2 diabetes, a study that provided potential warning signs of Addison's disease in patients with type 1 diabetes, and an analysis that showed dehydroepiandrosterone therapy may preserve bone muscle mass in older women.
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