Zobrazeno 1 - 2
of 2
pro vyhledávání: '"Liat Feraru Feldman"'
Autor:
David J. Sas, Daniella Magen, Wesley Hayes, Hadas Shasha-Lavsky, Mini Michael, Indra Schulte, Anne-Laure Sellier-Leclerc, Jiandong Lu, Ali Seddighzadeh, Bahru Habtemariam, Tracy L. McGregor, Kenji P. Fujita, Yaacov Frishberg, Justine Bacchetta, Véronique Baudouin, Rachel Becker-Cohen, Shimrit Tzvi Behr, Efrat Ben-Shalom, Maria Berdaguer, Detlef Bockenhauer, Pierre Cochat, Martin Coenen, Carl H. Cramer, Georges Deschênes, Claire Dossier, Emilie Doye, Liat Feraru Feldman, Maximilian Hohenadel, Florentia Kaguelidou, Irina Libinson Zebegret, John C. Lieske, Anne Maisin, Dawn S. Milliner, Moran Plonsky Toder, Shirley Pollack, Aurélie Portefaix, Bruno Ranchin, Choni Rinat, Adnan Safdar, Gesa Schalk, Poyyapakkam R. Srivaths, Cheryl L. Tran, William Van't Hoff, Jenny Weinbrand-Goichberg, Irith Weissman
Publikováno v:
Genetics in Medicine. 24:654-662
Primary hyperoxaluria type 1 (PH1) is a rare, progressive, genetic disease with limited treatment options. We report the efficacy and safety of lumasiran, an RNA interference therapeutic, in infants and young children with PH1.This single-arm, open-l
Publikováno v:
Harefuah. 154(6)
Endocarditis is an uncommon presentation of Kingella kingae infection in children. A previously healthy 17 month old child was referred to our emergency department for evaluation of fever lasting eleven days, aphthous stomatitis and a new systolic mu