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Journal Articles

Evaluation of in Vitro Macrophage Characterization in Gaucher Type 1 (GD1) Patients

Paola Delbini, PhD, Viola Ghiandai, Maria Domenica Cappellini, MD, Lorena Duca, Isabella Nava, Elena Cassinerio, MD, Irene Motta, MD

Blood (2019) 134 (Supplement_1): 3592.

https://doi.org/10.1182/blood-2019-130032

Published: 2019

Journal Articles

Computational Analysis Model Applied to Spanish Gaucher Registry Data

Marcio M Andrade-Campos, MD PhD, Laura Lopez de Frutos, PhD, Jorge J Cebolla, MSc, Blanca Medrano-Engay, PhD, Mercedes Roca-Espiau, PhD, Beatriz Gomez-Barrera, David Iniguez, Pilar Giraldo, PhD

Blood (2019) 134 (Supplement_1): 2323.

https://doi.org/10.1182/blood-2019-122347

Published: 2019

Journal Articles

Biomarker Response to Oral Eliglustat in Adults with Gaucher Disease Type 1: Results from 4 Completed Clinical Trials

Neal J Weinreb, MD, Timothy M Cox, MD, Pramod K. Mistry, MD PhD FRCP, Joel Charrow, MD, Elena Lukina, MD PhD Professor, Meredith C. Foster, ScD, M. Judith Peterschmitt, MD

Blood (2019) 134 (Supplement_1): 4859.

https://doi.org/10.1182/blood-2019-127337

Published: 2019

Journal Articles

Type 1 Gaucher Disease Severity and Risk for Liver Fibrosis in Untreated Patients and in Patients Treated Primarily with Enzyme Replacement Therapy for a Median of 20 Years

Neal J Weinreb, MD, Robert A Ali, MD

Blood (2018) 132 (Supplement 1): 2403.

https://doi.org/10.1182/blood-2018-99-117215

Published: 2018

Journal Articles

Repair of the Blood Brain Barrier and Neutrophil Recovery Following HSCT in Cerebral Adrenoleukodystrophy

Troy C Lund, MDMS, PhD, Weston P. Miller, MD, Dave Nascene, MD, Paul Orchard, MD, Ashish O. Gupta, MBBS, MPH

Blood (2018) 132 (Supplement 1): 4628.

https://doi.org/10.1182/blood-2018-99-109801

Published: 2018

Journal Articles

Correlation between Bone Disease and Genotype in Patients with Type 1 Gaucher Disease. Data from the Spanish Gaucher Disease Registry

Marcio Andrade-Campos, PhD, Pilar Irun, PhD, Mercedes Roca-Espiau, PhD, Pilar Giraldo, PhD

Blood (2017) 130 (Supplement 1): 3571.

https://doi.org/10.1182/blood.V130.Suppl_1.3571.3571

Published: 2017

Journal Articles

Impact of Velaglucerase Alfa in the Inflammatory Status of Type 1 Gaucher Disease Patients after One Year on Therapy. A Prospective Multicentre Study

Marcio Andrade-Campos, MD, Javier Gervas, PhD, Inmaculada García-Jiménez, MD, Olga Salamero, Pedro Martinez-Odriozola, MD, Jose Angel Mendez, MD, Fernando Garcia-Bragado, MD, Cristina Fernandez-Canal, MD, Luis Ignacio Sancho-Val, PhD, Horacio Cano, MD, Jordi Perez-Lopez, MD, Jesus Maria Hernandez-Rivas, PhD, Juan Francisco Lorenzo, MD, Miguel Lopez-Dupla, MD, Virginia Callao, PhD, Pilar Giraldo, PhD

Blood (2015) 126 (23): 4606.

https://doi.org/10.1182/blood.V126.23.4606.4606

Published: 2015

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

Blood (2014) 124 (21): 4116.

https://doi.org/10.1182/blood.V124.21.4116.4116

Published: 2014

Journal Articles

Long Term Outcomes of ERT in Children with Gaucher Disease. Spanish Experience

Marcio M Andrade, MD, Pilar Alfonso, PhD, Pilar Irún, PhD, Javier Dalmau, MD, Jose Luis Barbera, MD, Horacio Cano, MD, María Angeles Fernandez-Galan, MD, Rafael Franco, MD, Inmaculada Garcia, MD, Angela IbaÃ±ez, MD, Francisco Lendinez, MD, Elena Martin-Hernandez, MD, Luis Antonio Sancho-Val, MD, Pablo Sanjurjo, MDPhD, Miguel Pocovi, Prof, Pilar Giraldo, MD PhD

Blood (2014) 124 (21): 4950.

https://doi.org/10.1182/blood.V124.21.4950.4950

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.

Blood (2012) 120 (21): 1038.

https://doi.org/10.1182/blood.V120.21.1038.1038

Published: 2012

Journal Articles

Replacement Therapy with Imiglucerase Improves Magnetic Resonance Imaging (MRI) Abnormalities in Almost Half Patients with Non-Neuronopathic Form of Gaucher Disease After Twelve Months of Therapy: Results of a Semi-Quantitative MRI Method.

Veroniki Komninaka, Dionysia Kolomodi, Theodore Marinakis, Konstantina Repa, Dimitrios Christoulas, Ersi Voskaridou, Meletios A Dimopoulos, MD, Evangelos Terpos

Blood (2012) 120 (21): 2132.

https://doi.org/10.1182/blood.V120.21.2132.2132

Published: 2012

Journal Articles

A Multicenter, Double-Blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Patients with Gaucher Disease.

Ari Zimran, MD, Derlis Emilio Gonzalez-Rodriguez, MD, Aya Abrahamov, MD, Deborah Elstein, PhD, Rene Heitner, MD, Alona Paz, MD, Einat Brill-Almon, PhD, Raul Chertkoff, MD

Blood (2012) 120 (21): 2140.

https://doi.org/10.1182/blood.V120.21.2140.2140

Published: 2012

Journal Articles

Miglustat Therapy in Type 1 Gaucher Disease: Long-Term Treatment Experience From a Multicenter, Retrospective Cohort Study,

David J. Kuter, MD, DPhil, Atul Mehta, MD, Carla Hollak, MD, PhD, Pilar Giraldo, MD, PhD, Derralynn Hughes, MRCP, MRCPath, DPhil, MA, Nadia Belmatoug, MD, Monika Brand, Audrey Muller, Berthold Schaaf, Ruben Giorgino, Ari Zimran, MD

Blood (2011) 118 (21): 3207.

https://doi.org/10.1182/blood.V118.21.3207.3207

Published: 2011

Journal Articles

Bone Involvement in Patients with Non-Neuronopathic Gaucher Disease Is Characterized by Increased Osteoclast Function Partially Due to High Circulating C-C Motif Ligand 3,

Veroniki Komninaka, Theodore Marinakis, Dimitrios Christoulas, Konstantina Repa, Dionysia Kolomodi, Maria Mitropoulou, Maria Gkotzamanidou, Athanasios Papatheodorou, Nikolaos Anagnostopoulos, Meletios Athanasios Dimopoulos, MD, Evangelos Terpos

Blood (2011) 118 (21): 3215.

https://doi.org/10.1182/blood.V118.21.3215.3215

Published: 2011

Journal Articles

The Uptake of Recombinant Glucocerebrosidases by Blood Monocytes From Type 1 Gaucher Disease Patients Is Heterogeneous

Juliette Berger, Jérome Stirnemann, MD, PhD, Céline Bourgne, Bruno Pereira, Pascale Pigeon, Djazia Heraoui, Chantal Rapatel, Christian Rose, MD, PhD, Nadia Belmatoug, MD, Marc G Berger, MD, PhD

Blood (2011) 118 (21): 1102.

https://doi.org/10.1182/blood.V118.21.1102.1102

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.

Blood (2011) 118 (21): 1104.

https://doi.org/10.1182/blood.V118.21.1104.1104

Published: 2011

Journal Articles

Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response

Nick Dekker, Laura van Dussen, Carla E. M. Hollak, Herman Overkleeft, Saskia Scheij, Karen Ghauharali, Mariëlle J. van Breemen, Maria J. Ferraz, Johanna E. M. Groener, Mario Maas, Frits A. Wijburg, Dave Speijer, Anna Tylki-Szymanska, Pramod K. Mistry, Rolf G. Boot, Johannes M. Aerts

Blood (2011) 118 (16): e118–e127.

https://doi.org/10.1182/blood-2011-05-352971

Published: 2011

Includes: Supplemental data

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

Blood (2010) 116 (21): 3791.

https://doi.org/10.1182/blood.V116.21.3791.3791

Published: 2010

Journal Articles

Analysis of Spanish Experience During Imiglucerase Shortage

Pilar Giraldo, PhD, Pilar Irún, PhD, Pilar Alfonso, PhD, Jaime Dalmau, MD, MAngeles Fernández-Galán, MD, Antonio Figueredo, MD, Angela Ibañez, MD, Jesús M Hernández-Rivas, PhD, Elisa Luño, MD, Francisca Marín-Jimenez, Guillermo Martin-Nuñez, MD, Jorge Montserrat, MD, Javier De la Serna, MD, PhD, Lucia Villalon, MD, Miguel Pocovi, PhD

Blood (2010) 116 (21): 4725.

https://doi.org/10.1182/blood.V116.21.4725.4725

Published: 2010

Journal Articles

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

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

Blood (2010) 116 (6): 893–899.

https://doi.org/10.1182/blood-2010-03-273151

Published: 2010

NARROW

Evaluation of in Vitro Macrophage Characterization in Gaucher Type 1 (GD1) Patients

Computational Analysis Model Applied to Spanish Gaucher Registry Data

Biomarker Response to Oral Eliglustat in Adults with Gaucher Disease Type 1: Results from 4 Completed Clinical Trials

Type 1 Gaucher Disease Severity and Risk for Liver Fibrosis in Untreated Patients and in Patients Treated Primarily with Enzyme Replacement Therapy for a Median of 20 Years

Repair of the Blood Brain Barrier and Neutrophil Recovery Following HSCT in Cerebral Adrenoleukodystrophy

Correlation between Bone Disease and Genotype in Patients with Type 1 Gaucher Disease. Data from the Spanish Gaucher Disease Registry

Impact of Velaglucerase Alfa in the Inflammatory Status of Type 1 Gaucher Disease Patients after One Year on Therapy. A Prospective Multicentre Study

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

Long Term Outcomes of ERT in Children with Gaucher Disease. Spanish Experience

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

Replacement Therapy with Imiglucerase Improves Magnetic Resonance Imaging (MRI) Abnormalities in Almost Half Patients with Non-Neuronopathic Form of Gaucher Disease After Twelve Months of Therapy: Results of a Semi-Quantitative MRI Method.

A Multicenter, Double-Blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Patients with Gaucher Disease.

Miglustat Therapy in Type 1 Gaucher Disease: Long-Term Treatment Experience From a Multicenter, Retrospective Cohort Study,

Bone Involvement in Patients with Non-Neuronopathic Gaucher Disease Is Characterized by Increased Osteoclast Function Partially Due to High Circulating C-C Motif Ligand 3,

The Uptake of Recombinant Glucocerebrosidases by Blood Monocytes From Type 1 Gaucher Disease Patients Is Heterogeneous

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

Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response

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

Analysis of Spanish Experience During Imiglucerase Shortage

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

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American Society of Hematology

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American Society of Hematology

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