Not logged in Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial Michael, Mini; Groothoff, Jaap W.; Shasha-Lavsky, Hadas; Lieske, John C.; Frishberg, Yaacov; Simkova, Eva; Sellier-Leclerc, Anne-Laure; Devresse, Arnaud; Guebre-Egziabher, Fitsum; Bakkaloglu, Sevcan A.; Mourani, Chebl; Saqan, Rola; Singer, Richard; Willey, Richard; Habtemariam, Bahru; Gansner, John M.; Bhan, Ishir; McGregor, Tracy; Magen, Daniella Share Save
Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children Sas, David J.; Magen, Daniella; Hayes, Wesley; Shasha-Lavsky, Hadas; Michael, Mini; Schulte, Indra; Sellier-Leclerc, Anne-Laure; Lu, Jiandong; Seddighzadeh, Ali; Habtemariam, Bahru; McGregor, Tracy L.; Fujita, Kenji P.; Frishberg, Yaacov Share Save
Randomized Clinical Trial on the Long-Term Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1 Hulton, Sally A.; Groothoff, Jaap W.; Frishberg, Yaacov; Koren, Michael J.; Overcash, J. Scott; Sellier-Leclerc, Anne-Laure; Shasha-Lavsky, Hadas; Saland, Jeffrey M.; Hayes, Wesley; Magen, Daniella; Moochhala, Shabbir H.; Coenen, Martin; Simkova, Eva; Garrelfs, Sander F.; Sas, David J.; Meliambro, Kristin A.; Ngo, Taylor; Sweetser, Marianne T.; Habtemariam, Bahru A.; Gansner, John M.; McGregor, Tracy L.; Lieske, John C. Share Save
Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1 A Placebo-Controlled Randomized Clinical Trial Frishberg, Yaacov; Deschenes, Georges; Groothoff, Jaap W.; Hulton, Sally-Anne; Magen, Daniella; Harambat, Jerome; van't Hoff, William G.; Lorch, Ulrike; Milliner, Dawn S.; Lieske, John C.; Haslett, Patrick; Garg, Pushkal P.; Vaishnaw, Akshay K.; Talamudupula, Sandeep; Lu, Jiandong; Habtemariam, Bahru A.; Erbe, David, V; McGregor, Tracy L.; Cochat, Pierre Share Save
Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1 Garrelfs, Sander F.; Frishberg, Yaacov; Hulton, Sally A.; Koren, Michael J.; O'Riordan, William D.; Cochat, Pierre; Deschenes, Georges; Shasha-Lavsky, Hadas; Saland, Jeffrey M.; van't Hoff, William G.; Fuster, Daniel G.; Magen, Daniella; Moochhala, Shabbir H.; Schalk, Gesa; Simkova, Eva; Groothoff, Jaap W.; Sas, David J.; Meliambro, Kristin A.; Lu, Jiandong; Sweetser, Marianne T.; Garg, Pushkal P.; Vaishnaw, Akshay K.; Gansner, John M.; McGregor, Tracy L.; Lieske, John C. Share Save
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Characterising a healthy adult with a rare HAO1 knockout to support a therapeutic strategy for primary hyperoxaluria McGregor, Tracy L.; Hunt, Karen A.; Yee, Elaine; Mason, Dan; Nioi, Paul; Ticau, Simina; Pelosi, Marissa; Loken, Perry R.; Finer, Sarah; Lawlor, Deborah A.; Fauman, Eric B.; Huang, Qin Qin; Griffiths, Christopher J.; MacArthur, Daniel G.; Trembath, Richard C.; Oglesbee, Devin; Lieske, John C.; Erbe, David, V; Wright, John; van Heel, David A. Share Save
End Points for Clinical Trials in Primary Hyperoxaluria Milliner, Dawn S.; McGregor, Tracy L.; Thompson, Aliza; Dehmel, Bastian; Knight, John; Rosskamp, Ralf; Blank, Melanie; Yang, Sixun; Fargue, Sonia; Rumsby, Gill; Groothoff, Jaap; Allain, Meaghan; West, Melissa; Hollander, Kim; Lowther, W. Todd; Lieske, John C. Share Save
Acute kidney injury risk-based screening in pediatric inpatients: a pragmatic randomized trial Van Driest, Sara L.; Wang, Li; McLemore, Michael F.; Bridges, Brian C.; Fleming, Geoffrey M.; McGregor, Tracy L.; Jones, Deborah P.; Shirey-Rice, Jana; Gatto, Cheryl L.; Gay, James C.; Byrne, Daniel W.; Weitkamp, Asli; Roden, Dan M.; Bernard, Gordon Share Save
Cicatricial Alopecia Research Foundation meeting, May 2016: Progress towards the diagnosis, treatment and cure of primary cicatricial alopecias Sundberg, John P.; Hordinsky, Maria K.; Bergfeld, Wilma; Lenzy, Yolanda M.; McMichael, Amy J.; Christiano, Angela M.; McGregor, Tracy; Stenn, Kurt S.; Sivamani, Raja K.; Pratt, Craig Herbert; King, Lloyd E., Jr. Share Save
Electronic health record-based predictive models for acute kidney injury screening in pediatric inpatients Wang, Li; McGregor, Tracy L.; Jones, Deborah P.; Bridges, Brian C.; Fleming, Geoffrey M.; Shirey-Rice, Jana; McLemore, Michael F.; Chen, Lixin; Weitkamps, Asli; Byrne, Daniel W.; Van Driest, Sara L. Share Save
Acute Kidney Injury Incidence in Noncritically Ill Hospitalized Children, Adolescents, and Young Adults: A Retrospective Observational Study McGregor, Tracy L.; Jones, Deborah P.; Wang, Li; Danciu, Ioana; Bridges, Brian C.; Fleming, Geoffrey M.; Shirey-Rice, Jana; Chen, Lixin; Byrne, Daniel W.; Van Driest, Sara L. Share Save
Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study Schwahn, Bernd C.; Van Spronsen, Francjan J.; Belaidi, Abdel A.; Bowhay, Stephen; Christodoulou, John; Derks, Terry G.; Hennermann, Julia B.; Jameson, Elisabeth; Koenig, Kai; McGregor, Tracy L.; Font-Montgomery, Esperanza; Santamaria-Araujo, Jose A.; Santra, Saikat; Vaidya, Mamta; Vierzig, Anne; Wassmer, Evangeline; Weis, Ilona; Wong, Flora Y.; Veldman, Alex; Schwarz, Guenter Share Save
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Recurrent Deletions and Reciprocal Duplications of 10q11.21q11.23 Including CHAT and SLC18A3 are Likely Mediated by Complex Low-Copy Repeats Stankiewicz, Pawel; Kulkarni, Shashikant; Dharmadhikari, Avinash V.; Sampath, Srirangan; Bhatt, Samarth S.; Shaikh, Tamim H.; Xia, Zhilian; Pursley, Amber N.; Cooper, M. Lance; Shinawi, Marwan; Paciorkowski, Alex R.; Grange, Dorothy K.; Noetzel, Michael J.; Saunders, Scott; Simons, Paul; Summar, Marshall; Lee, Brendan; Scaglia, Fernando; Fellmann, Florence; Martinet, Danielle; Beckmann, Jacques S.; Asamoah, Alexander; Platky, Kathryn; Sparks, Susan; Martin, Ann S.; Madan-Khetarpal, Suneeta; Hoover, Jacqueline; Medne, Livija; Bonnemann, Carsten G.; Moeschler, John B.; Vallee, Stephanie E.; Parikh, Sumit; Irwin, Polly; Dalzell, Victoria P.; Smith, Wendy E.; Banks, Valerie C.; Flannery, David B.; Lovell, Carolyn M.; Bellus, Gary A.; Golden-Grant, Kathryn; Gorski, Jerome L.; Kussmann, Jennifer L.; McGregor, Tracy L.; Hamid, Rizwan; Pfotenhauer, Jean; Ballif, Blake C.; Shaw, Chad A.; Kang, Sung-Hae L.; Bacino, Carlos A.; Patel, Ankita; Rosenfeld, Jill A.; Cheung, Sau Wai; Shaffer, Lisa G. Share Save
An Evolutionary Genomic Approach to Identify Genes Involved in Human Birth Timing Plunkett, Jevon; Doniger, Scott; Orabona, Guilherme; Morgan, Thomas; Haataja, Ritva; Hallman, Mikko; Puttonen, Hilkka; Menon, Ramkumar; Kuczynski, Edward; Norwitz, Errol; Snegovskikh, Victoria; Palotie, Aarno; Peltonen, Leena; Fellman, Vineta; DeFranco, Emily A.; Chaudhari, Bimal P.; McGregor, Tracy L.; McElroy, Jude J.; Oetjens, Matthew T.; Teramo, Kari; Borecki, Ingrid; Fay, Justin; Muglia, Louis Share Save