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Journal Articles

Bone Marrow Involvement in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long Term Enzyme Replacement Therapy (median 18 years)

Shoshana Revel-Vilk, MD, Jeffrey Szer, MBBS, Michal Becker Cohen, Ari Zimran, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4862.
https://doi.org/10.1182/blood-2019-131013
Published: 2019
Journal Articles

Liver-Directed AAV Gene Therapy for Gaucher Disease

Carlos J Miranda, PhD, Miriam Canavese, PhD, Elisa Chisari, MS, Jalpa Pandya, MPhil, Clement Cocita, PhD, Maria Portillo, MRes, Jenny McIntosh, PhD, Azadeh Kia, PhD, Jonathan H. Foley, PhD, Allison Dane, PhD, Jey M Jeyakumar, PhD, Rose Sheridan, PhD, Romuald Corbau, Amit C. Nathwani, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3354.
https://doi.org/10.1182/blood-2019-124280
Published: 2019
Journal Articles

Reduction in Intravenous Infusion Duration of Enzyme Replacement Therapy to Ten Minutes with Velaglucerase Alfa in Adult Patients with Type 1 Gaucher Disease

Ari Zimran, MD, Shoshana Revel-Vilk, MD, Deborah Elstein, PhD, Naama Arbel, Michal Becker-Cohen, MSc, Gaya Chicco, Jeff Szer, MB, BS, FRACP
Journal: Blood
Blood (2017) 130 (Supplement 1): 2101.
https://doi.org/10.1182/blood.V130.Suppl_1.2101.2101
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
Journal: Blood
Blood (2015) 126 (23): 4606.
https://doi.org/10.1182/blood.V126.23.4606.4606
Published: 2015
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
Journal: Blood
Blood (2014) 124 (21): 4963.
https://doi.org/10.1182/blood.V124.21.4963.4963
Published: 2014
Journal Articles

Persistence of Imiglucerase in Cell Compartment Explains Dosing Frequency of Every Two Weeks in Type 1 Gaucher Disease

Juliette Berger, PhD, Marie Vigan, PhD, Jérôme Stirnemann, PD, PhD Professor, Céline Bourgne, PhD, Alexandre Janel, PhD, Nadia Belmatoug, MD, Christian Rose, MD, Monia Bengherbia, Bruno Pereira, France Mentré, MD PhD Professor, Marc G Berger, MD PhD
Journal: Blood
Blood (2014) 124 (21): 1396.
https://doi.org/10.1182/blood.V124.21.1396.1396
Published: 2014
Journal Articles

Two-Year Safety and Tolerability of Velaglucerase Alfa in Patients with Type 1 Gaucher Disease, Including Patients Switched From Imiglucerase: Phase III Trial HGT-GCB-039 and Extension,

A Mehta, H Ben Turkia, DE Gonzalez, M Kabra, EA Lukina, P Giraldo, I Kisinovsky, A Bavdekar, M-F Ben Dridi, N Gupta, PS Kishnani, EK Sureshkumar, N Barton, N Wang, E Crombez, K Bhirangi, A Zimran
Journal: Blood
Blood (2011) 118 (21): 3214.
https://doi.org/10.1182/blood.V118.21.3214.3214
Published: 2011
Journal Articles

Report on the Safety of Velaglucerase Alfa Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease and the Transition From Clinic to Home Infusions During Treatment Protocol HGT-GCB-058

Gregory M Pastores, Barry E Rosenbloom, MD, Neal J Weinreb, Ozlem Goker-Alpan, Rebecca Mardach, Mark Lipson, Jennifer Ibrahim, Gabriel M Cohn, David Zahrieh, Pramod K Mistry
Journal: Blood
Blood (2011) 118 (21): 1101.
https://doi.org/10.1182/blood.V118.21.1101.1101
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
Journal: Blood
Blood (2011) 118 (21): 1102.
https://doi.org/10.1182/blood.V118.21.1102.1102
Published: 2011
Journal Articles

Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience

Clinical Trials & Observations
Ari Zimran, Gheona Altarescu, Mici Philips, Drorit Attias, Marina Jmoudiak, Maher Deeb, Nan Wang, Kiran Bhirangi, Gabriel M. Cohn, Deborah Elstein
Journal: Blood
Blood (2010) 115 (23): 4651–4656.
https://doi.org/10.1182/blood-2010-02-268649
Published: 2010