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Journal Articles

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

Clinical Trials & Observations
Timothy M. Cox, Guillermo Drelichman, Renata Cravo, Manisha Balwani, Thomas Andrew Burrow, Ana Maria Martins, Elena Lukina, Barry Rosenbloom, Ozlem Goker-Alpan, Nora Watman, Amal El-Beshlawy, Priya S. Kishnani, Maria Lucia Pedroso, Sebastiaan J. M. Gaemers, Regina Tayag, M. Judith Peterschmitt
Journal: Blood
Blood (2017) 129 (17): 2375–2383.
https://doi.org/10.1182/blood-2016-12-758409
Published: 2017
Journal Articles

New Prognostic Factors in the Assessment of Bone Complications in Gaucher Disease. Report from the Argentinian Group for Diagnostic and Treatment for Gaucher Disease

Guillermo I. Drelichman, MD, Nicolas Fernandez Escobar, MD, Nora F Basack, MD, Maria S Larroude, MD, Gabriel Aguilar, MD, Hugo Robledo, MD, Leonardo J Feldman, MD, Graciela Elena, MD, Nora Watman, MD, Moira Bolesina, MD, Juan Jose Chain, MD, Paola Reichel, MD, Alba A Ruiz, MD, Maria F Cuello, MD, Alcira B Fynn, MD, Marcela A Aznar, MD, Marta Dragosky, MD, Norberto B Guelbert, MD, Adriana Arizo, MD, Graciela A Pujal, MD, Katja G Muller, MD
Journal: Blood
Blood (2014) 124 (21): 4116.
https://doi.org/10.1182/blood.V124.21.4116.4116
Published: 2014
Journal Articles

Clinical Characteristics and Prognosis in South-American Patients with Myelodysplastic Syndrome: A Multicenter Study

Carolina Belli, PhD, Ronald Feitosa Pinheiro, MD PhD, Maria Gabriela Flores, MD, Silvia M. M. Magalhaes, MD, Laura Kornblihtt, MD PhD, Alicia Enrico, MD, Juliana Cordeiro, Ana Lisa Basquiera, MD, Marcela Cavalcante de Andrade Silva, Roberta da Silva Tanizawa, Noemi Pintos, MD, Graciela Alfonso, MD, Jorge Arbelbide, MD, Francisco Sakamoto, MD, Nora Watman, MD, Ximena Valladares Ticona, Irene B Larripa, PhD, Soledad S. Undurraga, MD, Elvira RP Velloso, MD PhD
Journal: Blood
Blood (2014) 124 (21): 4651.
https://doi.org/10.1182/blood.V124.21.4651.4651
Published: 2014
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
Journal Articles

Cytogenetic Features and Prognosis In Argentinean Patients with Myelodysplastic Syndrome: a Multicenter Study

Carolina Belli, PhD, Raquel Bengió, MD, Pedro Negri Aranguren, MD, Francisco Sakamoto, MD, María Gabriela Flores, MD, Nora Watman, MD, Elsa Nucifora, MD, Jorge Arbelbide, MD, María Virginia Prates, MD, Irene B Larripa, PhD
Journal: Blood
Blood (2010) 116 (21): 1888.
https://doi.org/10.1182/blood.V116.21.1888.1888
Published: 2010
Journal Articles

Eliglustat Tartrate, a Novel Investigational Oral Substrate Reduction Therapy for Gaucher Disease Type 1: Updated Phase 2 Results.

Elena Lukina, Judith Peterschmitt, Nora Watman, Marta Dragosky, Marcelo Iastrebner, Gregory Pastores, Elsa Avila Arreguin, Hanna Rosenbaum, Mici Phillips, Mathilde Kaper, Arshadul Haque, Ana Cristina Puga
Journal: Blood
Blood (2010) 116 (21): 3791.
https://doi.org/10.1182/blood.V116.21.3791.3791
Published: 2010
Journal Articles

Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study

Brief Report
Elena Lukina, Nora Watman, Elsa Avila Arreguin, Marta Dragosky, Marcelo Iastrebner, Hanna Rosenbaum, Mici Phillips, Gregory M. Pastores, Ravi S. Kamath, Daniel I. Rosenthal, Mathilde Kaper, Tejdip Singh, Ana Cristina Puga, M. Judith Peterschmitt
Journal: Blood
Blood (2010) 116 (20): 4095–4098.
https://doi.org/10.1182/blood-2010-06-293902
Published: 2010
Journal Articles

A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1

Clinical Trials & Observations
Elena Lukina, Nora Watman, Elsa Avila Arreguin, Maryam Banikazemi, Marta Dragosky, Marcelo Iastrebner, Hanna Rosenbaum, Mici Phillips, Gregory M. Pastores, Daniel I. Rosenthal, Mathilde Kaper, Tejdip Singh, Ana Cristina Puga, Peter L. Bonate, M. Judith Peterschmitt
Journal: Blood
Blood (2010) 116 (6): 893–899.
https://doi.org/10.1182/blood-2010-03-273151
Published: 2010