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     132  0 Kommentare Dicerna to Present Nedosiran Data at American Society of Nephrology (ASN) Kidney Week 2020

    Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that clinical data on nedosiran, an investigational candidate for the treatment of primary hyperoxaluria (PH), will be presented during the American Society of Nephrology (ASN) Kidney Week taking place Oct. 22-25, 2020. In addition, a commercial claims analysis of clinical and economic burden associated with PH will also be presented.

    The following poster presentations will be available during the “Genetic Diseases of the Kidneys: Non-Cystic – 1” session taking place on Thursday, Oct. 22, 2020 at 10:00 a.m. ET:

    • Title: PHYOX3: A Long-Term, Open-Label Extension Trial of Nedosiran in Patients With Primary Hyperoxaluria Type 1, 2 or 3
      Poster #: PO1625
    • Title: Clinical and Economic Impact of Primary Hyperoxaluria: A Retrospective Claims Analysis
      Poster #: PO1627

    About Primary Hyperoxaluria

    Primary hyperoxaluria (PH) is a family of ultra-rare, life-threatening genetic disorders that initially manifest with complications in the kidneys. There are three known types of PH (PH1, PH2 and PH3), each resulting from a mutation in one of three different genes. These genetic mutations cause enzyme deficiencies that result in the overproduction of a substrate called oxalate. Abnormal production and accumulation of oxalate leads to recurrent kidney stones, nephrocalcinosis and chronic kidney disease that may progress to end-stage renal disease, requiring intensive dialysis. Compromised renal function eventually results in the accumulation of oxalate in organs ranging from skin, bones, eyes and heart. In the most severe cases, symptoms start in the first year of life. A combined liver-kidney transplantation may be undertaken to resolve PH1 or PH2, but it is an invasive solution with limited availability and high morbidity that requires lifelong immune suppression to prevent organ rejection. There are currently no approved medications for the treatment of PH. Patients are limited to using hyperhydration and medication to attempt to increase solubility of oxalate in urine. Despite these interventions, oxalate may continue to accumulate in the kidneys and other organs, causing damage.

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    Dicerna to Present Nedosiran Data at American Society of Nephrology (ASN) Kidney Week 2020 Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that clinical data on nedosiran, an investigational candidate for …