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Journal Articles

Bone Marrow Plasma Cell Burden in Gaucher Disease Correlates with Local Macrophage Infiltration and May be Altered By Disease Treatment

Monika Klimkowska, MD PhD, Maciej J. Machaczka, MD PhD

Blood (2019) 134 (Supplement_1): 3593.

https://doi.org/10.1182/blood-2019-131343

Published: 2019

Journal Articles

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Bernhard Gentner, MD, Maria Ester Bernardo, MD PhD, Francesca Tucci, MD, Erika Zonari, PhD, Francesca Fumagalli, MD, Silvia Pontesilli, MD, Serena Acquati, PhD, Paolo Silvani, MD, Fabio Ciceri, MD, Attilio Rovelli, MD, Giancarlo La Marca, PhD, Rossella Parini, MD, Silvia Gregori, PhD, Eugenio Montini, PhD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD

Blood (2019) 134 (Supplement_1): 607.

https://doi.org/10.1182/blood-2019-128805

Published: 2019

Journal Articles

Induction of Activated T Follicular Helper Cells Is Critical for Anti-FVIII Inhibitor Development in Hemophilia a Mice

Weiqing Jing, MD PhD, Juan Chen, PhD, Yuanhua Cai, MD, Yingyu Chen, PhD, Jocelyn A. Schroeder, Weiguo Cui, MD PhD, Qizhen Shi, MD PhD

Blood (2019) 134 (Supplement_1): 92.

https://doi.org/10.1182/blood-2019-123777

Published: 2019

Journal Articles

Not All Patients with Mild Phenotype of Gaucher Disease (GD) Need Disease Specific Treatment As Reflected By Patients Reported Outcomes Measures

Shoshana Revel-Vilk, MD, Tama Dinur, PhD, Majdolen Istaiti, Dafna Frydman, DMV, Michal Becker-Cohen, MSc, Ari Zimran, MD

Blood (2019) 134 (Supplement_1): 4712.

https://doi.org/10.1182/blood-2019-123000

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

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

Blood (2019) 134 (Supplement_1): 4862.

https://doi.org/10.1182/blood-2019-131013

Published: 2019

Journal Articles

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Blood (2019) 134 (Supplement_1): 3345.

https://doi.org/10.1182/blood-2019-123432

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

Blood (2019) 134 (Supplement_1): 3354.

https://doi.org/10.1182/blood-2019-124280

Published: 2019

Journal Articles

Open-Label, Expanded Access Study of Taliglucerase Alfa in Patients with Gaucher Disease Requiring Enzyme Replacement Therapy

David J. Kuter, MD DPhil, Michael Wajnrajch, MD MPA, Betina Hernandez, MD, Rong Wang, MPH, Raul Chertkoff, MD MBA, Ari Zimran, MD

Blood (2018) 132 (Supplement 1): 3755.

https://doi.org/10.1182/blood-2018-99-112660

Published: 2018

Journal Articles

Combination of TREC Measurement and Next-Generation Sequencing in Newborn Screening for Severe Combined Immunodeficiency: A Pilot Program in Japan

Hideki Muramatsu, MD PhD, Daiei Kojima, MD, Yusuke Okuno, MD PhD, Shinsuke Kataoka, MD, Yoko Nakajima, Tetsuya Ito, Ikuya Tsuge, Sakai Yoshimi, Tomoaki Kato, Seiji Kojima, MD PhD, Yoshiyuki Takahashi, MD, PhD

Blood (2018) 132 (Supplement 1): 3717.

https://doi.org/10.1182/blood-2018-99-118261

Published: 2018

Journal Articles

Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four Completed Clinical Trials

Pramod K. Mistry, MD PhD FRCP, Joel Charrow, MD, Timothy Cox, MD, Elena Lukina, MD PhD, Theodore Marinakis, MD, Meredith C. Foster, ScD, Sebastiaan J.M. Gaemers, MD, Judith Peterschmitt, MD

Blood (2018) 132 (Supplement 1): 2396.

https://doi.org/10.1182/blood-2018-99-117289

Published: 2018

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

MDS/Secondary AML Associated with Type I Gaucher Disease

Atsushi Okamoto, MD, Kenta Yamamoto, Go Eguchi, Yoshitaka Kanai, Terufumi Yamaguchi, Yasuhiro Maeda, MD PhD

Blood (2018) 132 (Supplement 1): 5193.

https://doi.org/10.1182/blood-2018-99-116058

Published: 2018

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

Early Diagnosis of an Infant with Gaucher's Disease Type 3. Case Report

Adriana C. Bello, MD, Rossana Cortez

Blood (2018) 132 (Supplement 1): 4950.

https://doi.org/10.1182/blood-2018-99-110263

Published: 2018

Journal Articles

Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice

Azadeh Kia, PhD, Jenny McIntosh, PhD, Cecilia Rosales, PhD, Paniz Hosseini, Rose Sheridan, Justyna Spiewak, Kevin Mills, Romuald Corbau, PhD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 2209.

https://doi.org/10.1182/blood-2018-99-116632

Published: 2018

Journal Articles

The Long-Term Adverse Event Profile of Oral Eliglustat for the Treatment of Gaucher Disease Type 1: Pooled Analysis of Data from 393 Patients in 4 Completed Trials

M. Judith Peterschmitt, MD, Selena Freisens, MD, Audrey W Hou, PharmD, Lisa Underhill, MS, Meredith C. Foster, ScD, Sebastiaan J.M. Gaemers, MD

Blood (2018) 132 (Supplement 1): 3692.

https://doi.org/10.1182/blood-2018-99-118319

Published: 2018

Journal Articles

In Vitro Erythropoietic Response to Enzyme Replacement Therapy in Gaucher Type 1 Patients

Paola Delbini, PhD, Irene Motta, Elena Cassinerio, Lorena Duca, Elena Trombetta, Margherita Migone De Amicis, Maria Domenica Cappellini, MD

Blood (2017) 130 (Supplement 1): 3476.

https://doi.org/10.1182/blood.V130.Suppl_1.3476.3476

Published: 2017

Journal Articles

Preliminary Results of Gau-PED Study: Prevalence of Gaucher Disease in Paediatric Patients Selected By an Appropriate Diagnostic Algorithm

William Morello, MD, Mirella Filocamo, Marina Stroppiano, Maja Di Rocco, MD, Nicola Tovaglieri, MD, Giovanna Russo, MD, Francesca Furlan, MD, Roberta Burnelli, MD, Andrea Pession, MD

Blood (2017) 130 (Supplement 1): 1187.

https://doi.org/10.1182/blood.V130.Suppl_1.1187.1187

Published: 2017

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

Blood (2017) 130 (Supplement 1): 2101.

https://doi.org/10.1182/blood.V130.Suppl_1.2101.2101

Published: 2017

NARROW

Bone Marrow Plasma Cell Burden in Gaucher Disease Correlates with Local Macrophage Infiltration and May be Altered By Disease Treatment

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Induction of Activated T Follicular Helper Cells Is Critical for Anti-FVIII Inhibitor Development in Hemophilia a Mice

Not All Patients with Mild Phenotype of Gaucher Disease (GD) Need Disease Specific Treatment As Reflected By Patients Reported Outcomes Measures

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

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

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Liver-Directed AAV Gene Therapy for Gaucher Disease

Open-Label, Expanded Access Study of Taliglucerase Alfa in Patients with Gaucher Disease Requiring Enzyme Replacement Therapy

Combination of TREC Measurement and Next-Generation Sequencing in Newborn Screening for Severe Combined Immunodeficiency: A Pilot Program in Japan

Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four 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

MDS/Secondary AML Associated with Type I Gaucher Disease

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

Early Diagnosis of an Infant with Gaucher's Disease Type 3. Case Report

Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice

The Long-Term Adverse Event Profile of Oral Eliglustat for the Treatment of Gaucher Disease Type 1: Pooled Analysis of Data from 393 Patients in 4 Completed Trials

In Vitro Erythropoietic Response to Enzyme Replacement Therapy in Gaucher Type 1 Patients

Preliminary Results of Gau-PED Study: Prevalence of Gaucher Disease in Paediatric Patients Selected By an Appropriate Diagnostic Algorithm

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

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American Society of Hematology

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