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Mathilde Kaper
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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
Journal Articles

Genz-112638 for Gaucher Disease Type 1: Phase 2 Clinical Trial Results After 18 Months of Treatment.

Judith Peterschmitt, MD, Elena Lukina, PhD, MD, Nora Patricia Watman, MD, Elsa Avila Arreguin, MD, Maryam Banikazemi, MD, Gregory Pastores, MD, Marcelo Iastrebner, MD, Marta Dragosky, MD, Hanna Rosenbaum, MD, Mici Phillips, MD, Mathilde Kaper, Tejdip Singh, MD, Ana Cristina Puga, MD, PhD
Journal: Blood
Blood (2009) 114 (22): 1349.
https://doi.org/10.1182/blood.V114.22.1349.1349
Published: 2009