Zoe Webster, Lisa Woods, Robert Dalziel et al.
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Condensed from the publisher's abstract. An xxcode editorial summary of this publication has not been generated yet.
Ciliopathies represent a diverse group of inherited disorders resulting from dysfunctional cilia. A rare subset is associated with biallelic variants in WDR19 , with fewer than 100 affected individuals reported worldwide.
Published in Hum Mutat. The text above is extracted from the publisher's own abstract and has not been edited by xxcode. For clinical decisions, review the original publication.
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This multicenter, phase 3 randomized trial compared frontline ponatinib plus blinatumomab with imatinib plus chemotherapy in adults with Philadelphia-positive acute lymphoblastic leukemia.
RISE UP was a phase 3, double-blind, global, randomised, placebo-controlled trial conducted at 69 sites in 15 countries. It evaluated twice-daily mitapivat in patients older than 16 years with sickle cell disease…
Two 52-week, double-blind, randomized, placebo-controlled phase III trials evaluated monthly or less frequent ianalumab in patients with active Sjögren's disease.
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