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Journal Articles

Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study

Clinical Trials & Observations
Jong Wook Lee, Flore Sicre de Fontbrune, Lily Wong Lee Lee, Viviani Pessoa, Sandra Gualandro, Wolfgang Füreder, Vadim Ptushkin, Scott T. Rottinghaus, Lori Volles, Lori Shafner, Rasha Aguzzi, Rajendra Pradhan, Hubert Schrezenmeier, Anita Hill
Journal: Blood
Blood (2019) 133 (6): 530–539.
https://doi.org/10.1182/blood-2018-09-876136
Published: 2019
Includes: Supplemental data
Journal Articles

Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria: Pharmacokinetics and Pharmacodynamics Observed in Two Phase 3 Randomized, Multicenter Studies

Regis Peffault De Latour, Robert A. Brodsky, MD, Stephan Ortiz, RPh, PhD, Antonio M. Risitano, MD PhD, Jun-Ho Jang, MD PhD, Peter Hillmen, MB ChB, PhD FRCP, FRCPath, Aleksandr Kulagin, Rajendra Pradhan, PhD, Scott T. Rottinghaus, MD, Rasha Aguzzi, MS, Richard A. Wells, MD DPhil, Jeffrey Szer, MBBS
Journal: Blood
Blood (2018) 132 (Supplement 1): 626.
https://doi.org/10.1182/blood-2018-99-110858
Published: 2018
Journal Articles

A Phase 3 Study of Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria Naive to Complement Inhibitors: Results of a Subgroup Analysis with Patients Stratified By Baseline Hemolysis Level, Transfusion History, and Demographics

Ilene C. Weitz, MD, Aleksandr Kulagin, Shinji Nakao, MD PhD, Caroline I. Piatek, MD, Jeffrey Szer, MBBS, Scott T. Rottinghaus, MD, Lori Volles, MD, Andrew I. Damokosh, PhD, Rasha Aguzzi, MS, Loree Larratt, MD, Antonio M. Risitano, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 627.
https://doi.org/10.1182/blood-2018-99-110623
Published: 2018
Journal Articles

Optimization of Dose Regimen for ALXN1210, a Novel Complement C5 Inhibitor, in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results of 2 Phase 1/2 Studies

Alexander Roeth, MD, Scott T. Rottinghaus, MD, Anita Hill, MBChB (Hons), PhD, Jin Seok Kim, MD PhD, Hubert Schrezenmeier, MD, Louis Terriou, MD, Alvaro Urbano-Ispizua, MD, Richard A. Wells, MD DPhil, Jun Ho Jang, MD PhD, Jeff Szer, MB, BS, FRACP, Rasha Aguzzi, MS, Eric Scott Bachman, MD PhD, Andrew I. Damokosh, PhD, Lori Shafner, PhD, Jong-Wook Lee, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 3482.
https://doi.org/10.1182/blood.V130.Suppl_1.3482.3482
Published: 2017
Journal Articles

Immediate, Complete, and Sustained Inhibition of C5 with ALXN1210 Reduces Complement-Mediated Hemolysis in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Interim Analysis of a Dose-Escalation Study

Jong-Wook Lee, MD, Eric Scott Bachman, MD PhD, Rasha Aguzzi, MS, Jun Ho Jang, MD PhD, Jin Seok Kim, MD PhD, Scott T. Rottinghaus, MD, Lori Shafner, PhD, Jeff Szer, MB, BS, FRACP
Journal: Blood
Blood (2016) 128 (22): 2428.
https://doi.org/10.1182/blood.V128.22.2428.2428
Published: 2016
Journal Articles

Eliglustat, an Investigational Oral Therapy for Gaucher Disease Type 1: Phase 2 Results After 4 Years of Treatment

M. Judith Peterschmitt, MD, Elena Lukina, PhD, MD, Nora Watman, MD, Marta Dragosky, M.D., Gregory M Pastores, Elsa Avila Arreguin, M.D., Hanna Rosenbaum, MD, Ari Zimran, MD, Elena Sysoeva, Rasha Aguzzi, Leorah H Ross, Ana Cristina Puga, M.D.
Journal: Blood
Blood (2012) 120 (21): 1038.
https://doi.org/10.1182/blood.V120.21.1038.1038
Published: 2012
Journal Articles

Improvements in Skeletal Manifestations in Gaucher Disease Type 1 Patients After 3 Years of Treatment with Oral Eliglustat During a Phase 2 Trial,

M. Judith Peterschmitt, MD, Ravi S Kamath, MD, Elena Lukina, PhD, MD, Nora Watman, MD, Marta Dragosky, M.D., Marcelo Iastrebner, M.D., Gregory M Pastores, Elsa Avila Arreguin, M.D., Mici Phillips, M.D., Hanna Rosenbaum, MD, Elena Sysoeva, Rasha Aguzzi, Leorah H Ross, Ana Cristina Puga, M.D., Daniel I Rosenthal, M.D.
Journal: Blood
Blood (2011) 118 (21): 3216.
https://doi.org/10.1182/blood.V118.21.3216.3216
Published: 2011
Journal Articles

Eliglustat, An Investigational Oral Therapy for Gaucher Disease Type 1: Phase 2 Results After 3 Years

M. Judith Peterschmitt, MD, Elena Lukina, PhD, MD, Nora Watman, MD, Marta Dragosky, M.D., Marcelo Iastrebner, M.D., Gregory Pastores, M.D., Elsa Avila Arreguin, M.D., Mici Phillips, M.D., Hanna Rosenbaum, MD, Elena Sysoeva, Rasha Aguzzi, Leorah H Ross, Ana Cristina Puga, M.D.
Journal: Blood
Blood (2011) 118 (21): 1104.
https://doi.org/10.1182/blood.V118.21.1104.1104
Published: 2011