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Journal Articles

Prospective National-Base Multicenter Study to Standardize the Follow-up of Type 1Gaucher Disease Patients Treated with Eliglustat Under Standard of Care Practice. Trazelga Project

Marcio M Andrade-Campos, PhD, Jorge J Cebolla, MSc, Laura Lopez de Frutos, PhD, Pilar Irun, PhD, Soledad Noya, MD, Santiago Nieto, MD, Lucia Villalon, PhD, Ana I Arribas, MD, Abelardo Barez, PhD, Javier Garcia-Frade, PD, Maria Lourdes Hermosin, MD, Jesús-María Hernández-Rivas, MD PhD, Daiana Ibarretxe, MD, Maria L Lozano, MD PhD, Teresa Molero, PhD, Maria Angeles Perez-Saenz, MDPhD, Jesus Villarrubia, PhD, Pilar Giraldo, PhD

Blood (2018) 132 (Supplement 1): 4942.

https://doi.org/10.1182/blood-2018-99-115534

Published: 2018

Journal Articles

Altering Glycosphingolipid Composition to Improve Multiple Myeloma Bone Complication

Houfu Leng, MSc, DPhil, MBBS, BSc, Adel Ersek, PhD, Emma Morris, DPhil, Beatriz Gamez Molina, PhD, Claire M. Edwards, PhD, Nicole Horwood, PhD

Blood (2018) 132 (Supplement 1): 1942.

https://doi.org/10.1182/blood-2018-99-112237

Published: 2018

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

Assessment of Hematologic and Visceral Parameters in Patients with Type 1 Gaucher Disease in Spain: An Epidemiological Study Using the Therapeutic Goals MAP (Monitor, Action and Progress) Tool ®

Pilar Giraldo, Jordi Perez, Ramiro Núñez, Rafael Fernandez de la Puebla, Elisa Luño, Salvador Saura, Juan Carlos Bureo, Javier de la Serna, Sylvia Plaza, Víctor Navas

Blood (2014) 124 (21): 4963.

https://doi.org/10.1182/blood.V124.21.4963.4963

Published: 2014

Journal Articles

Substrate Reduction Therapy With Miglustat In Type 1 Gaucher Disease In Spain. Nine Years Outcomes Update On ZAGAL Study

Pilar Giraldo, MD PhD, Marcio M Andrade, Sr., MD, Blanca Medrano, Pilar Alfonso, PhD, Pilar Irún, PhD, Koldo Atutxa, MD, Angeles Fernandez-Galan, MD, Abelardo Barez, MD, Rafael Franco, MD, Inmaculada Roig, Vicente Giner, MD, Lucia Villalon, MD, Elisa Martinez-Estefano, MD, Elisa Luño, MD, Ines Loyola, MD, Olga Salamero, Javier de la Serna, MD, PhD, Miguel Pocovi

Blood (2013) 122 (21): 4713.

https://doi.org/10.1182/blood.V122.21.4713.4713

Published: 2013

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

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

Clinical, Genetic Characteristics and Geographical Distribution of Gaucher Disease In Iberian Peninsulae and Islands

Pilar Giraldo, PhD, Pilar Alfonso, PhD, Pilar Irún, PhD, Amparo Chabas, Lluisa Villageliu, Daniel Grinberg, Clara Sa Miranda, Miguel Pocovi

Blood (2010) 116 (21): 4729.

https://doi.org/10.1182/blood.V116.21.4729.4729

Published: 2010

Journal Articles

The substrate is down: is the IV out?

Jeff Szer

Blood (2010) 116 (6): 860–861.

https://doi.org/10.1182/blood-2010-05-285163

Published: 2010

Journal Articles

Glucose Ceramide Synthase Inhibitors Inhibit Osteoclast Activation Induced by Myeloma-Derived and De Novo Synthesized Glycosphingolipids.

Ke Xu, MD, Aristotelis Antonopoulos, PhD, Emmanouil Spanoudakis, MD, PhD, Simon Parry, PhD, Aristeidis Chaidos, MD, PhD, Ming Hu, PhD, Terry Butters, PhD, Anne Dell, PhD, Amin Rahemtulla, MBBS, PhD, FRCP, Nikki Horwood, PhD, Anastasios Karadimitris, PhD, MRCP, FRCPath

Blood (2009) 114 (22): 424.

https://doi.org/10.1182/blood.V114.22.424.424

Published: 2009

Journal Articles

Care Practices in Gaucher Disease: Results of a French Study.

Belmatoug Nadia, Nathalie Guffon, Jérôme Stirnemann, Christine Serratrice, Alain Robert, Christian Rose, MD, Padrazzi Bruno, Pierre Kaminsky

Blood (2009) 114 (22): 4441.

https://doi.org/10.1182/blood.V114.22.4441.4441

Published: 2009

Journal Articles

Real-World Clinical Experience with Long-Term Miglustat Therapy in Type 1 Gaucher Disease: The ZAGAL Project

Pilar Giraldo, MD, PhD, Pilar Alfonso, MAngeles Fernandez-Galan, Guillermo Martín-Nuñez, Abelardo Barez, MD, Rafael Franco, Dora Alonso, Antonio Acedo, Alejandro Martin, Elvira Martinez-Estefano, Vanessa Roldan, Sol Serrano, Paz Latre, Miguel Pocovi

Blood (2008) 112 (11): 4638.

https://doi.org/10.1182/blood.V112.11.4638.4638

Published: 2008

Journal Articles

Changes in the Atherogenic Profile in Type 1 Gaucher Disease Patients after Substrate Reduction Therapy

Pilar Giraldo, MD, PhD, Jose Puzo, Pilar Alfonso, Pilar Irun, Miguel Pocovi

Blood (2008) 112 (11): 4645.

https://doi.org/10.1182/blood.V112.11.4645.4645

Published: 2008

Journal Articles

Diagnostic and Disease Management Algorithms for Gaucher Disease: A Guide for Haematologists

Pramod K Mistry, Maria Domenica Cappellini, Elena Lukina, Hayri Özsan, Sara Mach Pascual, Hanna Rosenbaum, Maria Helena Solano, Zachary Simon Spigelman, Jesús Villarrubia, Nora Patricia Watman, Gero Massenkeil

Blood (2008) 112 (11): 4648.

https://doi.org/10.1182/blood.V112.11.4648.4648

Published: 2008

Journal Articles

Short-Term Effect of Oral Treatment with Miglustat in Type 1 Gaucher Disease.

Pilar Giraldo, MD, PhD, Dora Alonso, MDPhD, M. Acedo, MD, Rafael Franco, MD, Alberto Barez, MD, Alejandro Corrales, MDPhD, D. Serrano, MD, Vanessa Roldan, MD, Paz Latre, MD, Pilar Alfonso, MDPhD, Miguel Pocovi, MDPhD

Blood (2005) 106 (11): 2235.

https://doi.org/10.1182/blood.V106.11.2235.2235

Published: 2005

Journal Articles

Balanced Oral Substrate Reduction Therapy for Patients with Mild to Moderate Gaucher’s Disease: Initial Results in Clinical Practice.

Derralynn A. Hughes, MA DPhil MRCP, Robert J. Baker, MSc, Linda Richfield, RGN RM, Alan Milligan, RGJ RGN, Rebecca Bruce, RGN, Atul B. Mehta, MD FRCP FRCPath

Blood (2004) 104 (11): 3820.

https://doi.org/10.1182/blood.V104.11.3820.3820

Published: 2004

Journal Articles

Allogeneic BMT Followed by Substrate Deprivation Therapy with N-butyldeoxynojirimycin in a Child with Tay-Sachs Disease.

Hans J.F.M. Jacobs, Michel M.A.A.P. Willemsen, Ph.D., M.D., Jacqueline J.J. Groot-Loonen, Ph.D. M.D., Ron R.A. Wevers, Ph.D. M.D., Peter P.M. Hoogerbrugge, Ph.D. M.D.

Blood (2004) 104 (11): 5158.

https://doi.org/10.1182/blood.V104.11.5158.5158

Published: 2004

NARROW

Prospective National-Base Multicenter Study to Standardize the Follow-up of Type 1Gaucher Disease Patients Treated with Eliglustat Under Standard of Care Practice. Trazelga Project

Altering Glycosphingolipid Composition to Improve Multiple Myeloma Bone Complication

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

Assessment of Hematologic and Visceral Parameters in Patients with Type 1 Gaucher Disease in Spain: An Epidemiological Study Using the Therapeutic Goals MAP (Monitor, Action and Progress) Tool ®

Substrate Reduction Therapy With Miglustat In Type 1 Gaucher Disease In Spain. Nine Years Outcomes Update On ZAGAL Study

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

Analysis of Spanish Experience During Imiglucerase Shortage

Clinical, Genetic Characteristics and Geographical Distribution of Gaucher Disease In Iberian Peninsulae and Islands

The substrate is down: is the IV out?

Glucose Ceramide Synthase Inhibitors Inhibit Osteoclast Activation Induced by Myeloma-Derived and De Novo Synthesized Glycosphingolipids.

Care Practices in Gaucher Disease: Results of a French Study.

Real-World Clinical Experience with Long-Term Miglustat Therapy in Type 1 Gaucher Disease: The ZAGAL Project

Changes in the Atherogenic Profile in Type 1 Gaucher Disease Patients after Substrate Reduction Therapy

Diagnostic and Disease Management Algorithms for Gaucher Disease: A Guide for Haematologists

Short-Term Effect of Oral Treatment with Miglustat in Type 1 Gaucher Disease.

Balanced Oral Substrate Reduction Therapy for Patients with Mild to Moderate Gaucher’s Disease: Initial Results in Clinical Practice.

Allogeneic BMT Followed by Substrate Deprivation Therapy with N-butyldeoxynojirimycin in a Child with Tay-Sachs Disease.

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American Society of Hematology

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