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A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem

Orphanet journal of rare diseases, 2024-07, Vol.19 (1), p.262-11, Article 262

2024

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Transformative effect of a Humanitarian Program for individuals affected by rare diseases: building support systems and creating local expertise

Orphanet journal of rare diseases, 2022-04, Vol.17 (1), p.87-87, Article 87

2022

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Management of neuronopathic Gaucher disease: Revised recommendations

Journal of inherited metabolic disease, 2009-10, Vol.32 (5), p.660-664

2009

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Outcome of type III Gaucher disease on enzyme replacement therapy: Review of 55 cases

Journal of inherited metabolic disease, 2007-11, Vol.30 (6), p.935-942

2007

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Management of neuronopathic Gaucher disease: A European consensus

Journal of inherited metabolic disease, 2001-06, Vol.24 (3), p.319-327

2001

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HSD126 Diagnosing Rare Diseases: Referral Behaviors of Physicians in the United States

Value in health, 2024-06, Vol.27 (6), p.S242-S242

2024

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PCR148 Patient Involvement in the Development of Outcomes Measures Specific to Neuronopathic Gaucher Disease Type 2 and Type 3 to Assess HRQoL Over the Disease Journey

Value in health, 2022-12, Vol.25 (12), p.S419-S419

2022

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HSD93 Towards Diagnosing Rare Diseases: Referral Behavior of Physicians in Europe

Value in health, 2023-12, Vol.26 (12), p.S311-S312

2023

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SA73 Addressing Unmet Needs of Patients With Neuronopathic Gaucher Disease Type 2 and Type 3: Creation of the GARDIAN Patient Registry

Value in health, 2022-12, Vol.25 (12), p.S497-S497

2022

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Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease

Blood cells, molecules, & diseases, 2018-02, Vol.68, p.203-208

2018

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Management of neuronopathic Gaucher disease: Revised recommendations: Neurological manifestations of IEM in children, dedicated to Robert Surtees

Journal of inherited metabolic disease, 2009, Vol.32 (5), p.660-664

2009

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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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Understanding patient and parent/caregiver perceptions on gene therapy in Gaucher disease: an international survey

Orphanet journal of rare diseases, 2023-01, Vol.18 (1), p.5-5, Article 5

2023

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A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative

Orphanet journal of rare diseases, 2023-07, Vol.18 (1), p.195-11, Article 195

2023

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A charitable access program for patients with lysosomal storage disorders in underserved communities worldwide

Orphanet journal of rare diseases, 2021-01, Vol.16 (1), p.8-8, Article 8

2021

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Measuring disease activity and patient experience remotely using wearable technology and a mobile phone app: outcomes from a pilot study in Gaucher disease

Orphanet journal of rare diseases, 2019-09, Vol.14 (1), p.212-212, Article 212

2019

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Transition of patients with Gaucher disease type 1 from pediatric to adult care: results from two international surveys of patients and health care professionals

Frontiers in pediatrics, 2024-08, Vol.12, p.1439236

2024

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A collaborative approach to address the unmet needs of patients with neuronopathic Gaucher disease type 2 and type 3, the creation of GARDIAN, a patient-led, patient-owned global registry

Molecular genetics and metabolism, 2023-02, Vol.138 (2), p.107058, Article 107058

2023

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A global disease patient registry for neuronopathic Gaucher

Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.S42-S42

2020

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The IGA calls for greater clarity and accuracy in the use of non-comparables for the treatment of Gaucher disease

Molecular genetics and metabolism, 2024-02, Vol.141 (2), p.107798, Article 107798

2024

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No patient should be left behind: Pilot project to expand lysosomal disorder diagnostics in Africa

Molecular genetics and metabolism, 2024-02, Vol.141 (2), p.107879, Article 107879

2024

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Neuronopathic Gaucher disease; patient association takes the lead to improve awareness and encourage collaborative working

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S39-S40

2018

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Regional manager program of the International Gaucher Alliance: No patient left behind

Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.S42-S42

2020

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Foreword

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

2015

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Foreword

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

2015

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A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem

Transformative effect of a Humanitarian Program for individuals affected by rare diseases: building support systems and creating local expertise

Management of neuronopathic Gaucher disease: Revised recommendations

Outcome of type III Gaucher disease on enzyme replacement therapy: Review of 55 cases

Management of neuronopathic Gaucher disease: A European consensus

HSD126 Diagnosing Rare Diseases: Referral Behaviors of Physicians in the United States

PCR148 Patient Involvement in the Development of Outcomes Measures Specific to Neuronopathic Gaucher Disease Type 2 and Type 3 to Assess HRQoL Over the Disease Journey

HSD93 Towards Diagnosing Rare Diseases: Referral Behavior of Physicians in Europe

SA73 Addressing Unmet Needs of Patients With Neuronopathic Gaucher Disease Type 2 and Type 3: Creation of the GARDIAN Patient Registry

Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease

Management of neuronopathic Gaucher disease: Revised recommendations: Neurological manifestations of IEM in children, dedicated to Robert Surtees

The definition of neuronopathic Gaucher disease

Understanding patient and parent/caregiver perceptions on gene therapy in Gaucher disease: an international survey

A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative

A charitable access program for patients with lysosomal storage disorders in underserved communities worldwide

Measuring disease activity and patient experience remotely using wearable technology and a mobile phone app: outcomes from a pilot study in Gaucher disease

Transition of patients with Gaucher disease type 1 from pediatric to adult care: results from two international surveys of patients and health care professionals

A collaborative approach to address the unmet needs of patients with neuronopathic Gaucher disease type 2 and type 3, the creation of GARDIAN, a patient-led, patient-owned global registry

A global disease patient registry for neuronopathic Gaucher

The IGA calls for greater clarity and accuracy in the use of non-comparables for the treatment of Gaucher disease

No patient should be left behind: Pilot project to expand lysosomal disorder diagnostics in Africa

Neuronopathic Gaucher disease; patient association takes the lead to improve awareness and encourage collaborative working

Regional manager program of the International Gaucher Alliance: No patient left behind

Foreword

Foreword

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