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How I treat Gaucher disease

Blood, 2011-08, Vol.118 (6), p.1463-1471

Zimran, Ari

2011

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Value of Glucosylsphingosine (Lyso-Gb1) as a Biomarker in Gaucher Disease: A Systematic Literature Review

International journal of molecular sciences, 2020-10, Vol.21 (19), p.7159

2020

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How we manage Gaucher Disease in the era of choices

British journal of haematology, 2018-08, Vol.182 (4), p.467-480

2018

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Gaucher disease types 1 and 3: Phenotypic characterization of large populations from the ICGG Gaucher Registry

American journal of hematology, 2015-07, Vol.90 (S1), p.S12-S18

2015

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High-Dose Ambroxol Therapy in Type 1 Gaucher Disease Focusing on Patients with Poor Response to Enzyme Replacement Therapy or Substrate Reduction Therapy

International journal of molecular sciences, 2023-04, Vol.24 (7), p.6732

2023

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Glucosylsphingosine is a highly sensitive and specific biomarker for primary diagnostic and follow-up monitoring in Gaucher disease in a non-Jewish, Caucasian cohort of Gaucher disease patients

PloS one, 2013-11, Vol.8 (11), p.e79732-e79732

2013

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The definition of neuronopathic Gaucher disease

Journal of inherited metabolic disease, 2020-09, Vol.43 (5), p.1056-1059

2020

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Glucosylsphingosine (lyso-Gb1) as a Biomarker for Monitoring Treated and Untreated Children with Gaucher Disease

International journal of molecular sciences, 2019-06, Vol.20 (12), p.3033

2019

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Impact of Long-Term Enzyme Replacement Therapy on Glucosylsphingosine (Lyso-Gb1) Values in Patients with Type 1 Gaucher Disease: Statistical Models for Comparing Three Enzymatic Formulations

International journal of molecular sciences, 2021-07, Vol.22 (14), p.7699

2021

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Accuracy of chitotriosidase activity and CCL18 concentration in assessing type I Gaucher disease severity. A systematic review with meta-analysis of individual participant data

Haematologica (Roma), 2021-02, Vol.106 (2), p.437-445

2021

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Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease

Orphanet journal of rare diseases, 2018-02, Vol.13 (1), p.36-36, Article 36

2018

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Gaucher Disease Diagnosis Using Lyso-Gb1 on Dry Blood Spot Samples: Time to Change the Paradigm?

International journal of molecular sciences, 2022-01, Vol.23 (3), p.1627

2022

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Brain-Derived Neurotrophic Factor (BDNF) Is Associated with Platelet Activity and Bleeding Tendency in Patients with Gaucher Disease

International journal of molecular sciences, 2022-11, Vol.23 (22), p.13982

2022

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GBA1-Associated Parkinson’s Disease Is a Distinct Entity

International journal of molecular sciences, 2024-07, Vol.25 (13), p.7102

2024

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A Comprehensive Assessment of Qualitative and Quantitative Prodromal Parkinsonian Features in Carriers of Gaucher Disease-Identifying Those at the Greatest Risk

International journal of molecular sciences, 2022-10, Vol.23 (20), p.12211

2022

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Patient reported outcome measures in a large cohort of patients with type 1 Gaucher disease

Orphanet journal of rare diseases, 2020-10, Vol.15 (1), p.1-284, Article 284

2020

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Contribution of Glucosylsphingosine (Lyso-Gb1) to Treatment Decisions in Patients with Gaucher Disease

International journal of molecular sciences, 2023-02, Vol.24 (4), p.3945

2023

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A synergistic effect of Ambroxol and Beta-Glucosylceramide in alleviating immune-mediated hepatitis: A novel immunomodulatory non-immunosuppressive formulation for treatment of immune-mediated disorders

Biomedicine & pharmacotherapy, 2020-12, Vol.132, p.110890, Article 110890

2020

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Simultaneous Bilateral Femoral Osteonecrosis in Gaucher Disease

Life (Basel, Switzerland), 2023-05, Vol.13 (5), p.1135

2023

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A plant-derived recombinant human glucocerebrosidase enzyme--a preclinical and phase I investigation

PloS one, 2009-03, Vol.4 (3), p.e4792

2009

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Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials

Orphanet journal of rare diseases, 2022-01, Vol.17 (1), p.9-9, Article 9

2022

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Real-Life Experience with Oral Eliglustat in Patients with Gaucher Disease Previously Treated with Enzyme Replacement Therapy

Journal of clinical medicine, 2022-10, Vol.11 (21), p.6265

2022

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Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4years of treatment

Blood cells, molecules, & diseases, 2014-12, Vol.53 (4), p.274-276

2014

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A Feasibility Open-Labeled Clinical Trial Using a Second-Generation Artificial-Intelligence-Based Therapeutic Regimen in Patients with Gaucher Disease Treated with Enzyme Replacement Therapy

Journal of clinical medicine, 2024-06, Vol.13 (11), p.3325

2024

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The Bone Biomarker of Quantitative Chemical Shift Imaging in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long-Term Enzyme Replacement Therapy

Journal of clinical medicine, 2023-03, Vol.12 (6), p.2220

2023

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How I treat Gaucher disease

Value of Glucosylsphingosine (Lyso-Gb1) as a Biomarker in Gaucher Disease: A Systematic Literature Review

How we manage Gaucher Disease in the era of choices

Gaucher disease types 1 and 3: Phenotypic characterization of large populations from the ICGG Gaucher Registry

High-Dose Ambroxol Therapy in Type 1 Gaucher Disease Focusing on Patients with Poor Response to Enzyme Replacement Therapy or Substrate Reduction Therapy

Glucosylsphingosine is a highly sensitive and specific biomarker for primary diagnostic and follow-up monitoring in Gaucher disease in a non-Jewish, Caucasian cohort of Gaucher disease patients

The definition of neuronopathic Gaucher disease

Glucosylsphingosine (lyso-Gb1) as a Biomarker for Monitoring Treated and Untreated Children with Gaucher Disease

Impact of Long-Term Enzyme Replacement Therapy on Glucosylsphingosine (Lyso-Gb1) Values in Patients with Type 1 Gaucher Disease: Statistical Models for Comparing Three Enzymatic Formulations

Accuracy of chitotriosidase activity and CCL18 concentration in assessing type I Gaucher disease severity. A systematic review with meta-analysis of individual participant data

Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease

Gaucher Disease Diagnosis Using Lyso-Gb1 on Dry Blood Spot Samples: Time to Change the Paradigm?

Brain-Derived Neurotrophic Factor (BDNF) Is Associated with Platelet Activity and Bleeding Tendency in Patients with Gaucher Disease

GBA1-Associated Parkinson’s Disease Is a Distinct Entity

A Comprehensive Assessment of Qualitative and Quantitative Prodromal Parkinsonian Features in Carriers of Gaucher Disease-Identifying Those at the Greatest Risk

Patient reported outcome measures in a large cohort of patients with type 1 Gaucher disease

Contribution of Glucosylsphingosine (Lyso-Gb1) to Treatment Decisions in Patients with Gaucher Disease

A synergistic effect of Ambroxol and Beta-Glucosylceramide in alleviating immune-mediated hepatitis: A novel immunomodulatory non-immunosuppressive formulation for treatment of immune-mediated disorders

Simultaneous Bilateral Femoral Osteonecrosis in Gaucher Disease

A plant-derived recombinant human glucocerebrosidase enzyme--a preclinical and phase I investigation

Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials

Real-Life Experience with Oral Eliglustat in Patients with Gaucher Disease Previously Treated with Enzyme Replacement Therapy

Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4years of treatment

A Feasibility Open-Labeled Clinical Trial Using a Second-Generation Artificial-Intelligence-Based Therapeutic Regimen in Patients with Gaucher Disease Treated with Enzyme Replacement Therapy

The Bone Biomarker of Quantitative Chemical Shift Imaging in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long-Term Enzyme Replacement Therapy

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