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    Islet Interleukin-1{beta} Immunoreactivity Is an Early Feature of Cystic Fibrosis That May Contribute to {beta}-Cell Failure

    Tuesday, March 20, 2018 -- OBJECTIVE Cystic fibrosis–related diabetes (CFRD) is a common complication of cystic fibrosis (CF), increasing patient morbidity and mortality. Poor understanding of CFRD pathogenesis limits the development of targeted therapies to treat and/or prevent the disease. The aim of this study was to evaluate islet pathology, specifically, inflammation, amyloid deposition, and endocrine cell composition in subjects with CF with diabetes and with CF without diabetes. RESEARCH DESIGN AND METHODS A retrospective analysis of archived pancreas tissue collected at autopsy was conducted using pancreas tissue from subjects with CF and diabetes (CFRD) (n = 18) and CF without diabetes (CF-no DM) (n = 17). Two cohorts of control non-CF subjects were identified, each matched to CFRD and CF-no DM subjects for

    Proteostasis' PTI-428 gets FDA orphan drug designation to treat cystic fibrosis

    Thursday, March 15, 2018 -- Proteostasis Therapeutics has secured orphan drug designation from the US Food and Drug Administration (FDA) for its PTI-428, which is a cystic fibrosis transmembrane conductance regulator (CFTR) amplifier drug candidate.