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Type Name Description Download
Video Webinar: Newborn screening New born screening is the process of testing new born babies for serious, but treatable, conditions, including Gaucher disease. Watch
IGA materials Best Practice Sharing Published leaflet on best practices for GD patients (2018) Download
IGA materials IGA's Articles of Association (2020) IGA official document Download
IGA materials IGA's Code of Practice (2019) Code of Practice on Relationships with the pharmaceutical industry Download
IGA materials IGA's Code of Practice Glossary (2018) An addendum to the IGA's Code of Practice Download
IGA materials IGA Volunteer Charter (2019) An IGA policy Download
IGA materials IGA's Strategic Plan (2021) IGA's updated Strategic Plan Download
IGA materials IGA Impact Report (2019) IGA's Impact Report for 2019 Download
IGA materials IGA's Regional Manager Programme Poster published at WORLD and ECRD 2020 Download
IGA materials nGD registry Poster presented at WORLD and ECRD 2020 Download
IGA materials Best Practice Sharing Poster presented at ECRD 20148 Download
Links to other useful sites European Patients Forum EPF is an umbrella organisation that works with patients’ groups in public health and health advocacy across Europe. Visit
Links to other useful sites EUPATI Check out the EUPATI website for patient expert training courses and toolkits. Visit
Links to other useful sites Orphanet Orphanet is a unique resource, gathering and improving knowledge on rare diseases so as to improve the diagnosis, care and treatment of patients with rare diseases. Visit
Video Webinar: Digital Health Technology Educational webinar focussed on the growing use of digital technology to capture patient data, empower patients to be more involved in their health journey. Watch
Video Webinar: AAV based Gene therapy Educational webinars on the latest developments in gene therapy focused on AAV-based gene therapy and Prevail Therapeutic's approach for nGDand Parkinson’s disease with a GBA mutation. Watch
Video Webinar: Ex vivo gene therapy for Type 1 Gaucher disease Educational webinars on the latest developments in gene therapy for Gaucher treatment, focused on AVROBIO's lentiviral investigational gene therapy. Watch
Video Webinar: IGD2020: Home Therapy The focus of this year's IGD campaign was home therapy, following the challenges some Gaucher patients are facing during the COVID19 pandemic. Watch
Video Webinar: EWGGD GD1 Clinical Guidelines A webinar to set the scene to explore what should be included in new international patient centric guidelines for Type 1 Gaucher disease. Watch
Video Webinar: Gaucher & Parkinson's disease - the early signs and risks to carriers A webinar exploring how researches have linked GD to a slightly increased risk of having Parkinson's disease later in life. Watch
Video Webinar: Home Therapy with EWGGD IGA initiates global discussion on home therapy for Gaucher patients Watch
Literature from patient groups Patient & Family Support Services (UK Gauchers Association) The UK Gauchers Association employs a Patient & Family Support Worker to help with any non-clinical advice and information. Download
Literature from patient groups GD3 transitioning to adult care booklet (UK Gauchers Association) This booklet was written by young adult GD3 patients to help others understand what to expect while going through transition into adult services Download
Literature from patient groups What is homecare (UK Gauchers Assocaition) Homecare is a service provided to patients to enable them to receive ERT treatment away from the hospital Download
Literature from patient groups Planning a family (UK Gauchers Assocaition) Planning a family or finding out you are pregnant can be a very exciting time - this is a resource to help think about all the things to consider Download
Literature from patient groups Gene therapy for GD1 (UK Gauchers Assocaition) A booklet written by G Massaro, SN Waddington & AA Rahim from UCL about gene therapy: the introduction of genetic material to cells of patients for therapeutic benefit Download
Literature from patient groups Živeti z Gaucherjevo boleznijo (Slovenian Gaucher Association) Living with Gaucher Disease Download
Literature from patient groups David Explores Gaucher disease An information booklet from the USA National Gaucher Foundation View
IGA materials Gaucher Disease Basic Information Booklet We have developed this booklet as a source of information for patients and their carers and to answer questions on Gaucher disease Download
Scientific papers The European Gaucher Alliance: a survey of member patient organisations’ activities, healthcare environments and concerns The survey provided a ‘snapshot’ of the situation for patients and families affected by Gaucher disease, helping the EGA direct its activities into areas of greatest need. View
Scientific papers The road to biosimilars in rare diseases Ongoing lessons from Gaucher disease View
Scientific papers The definition of neuronopathic Gaucher disease This new definition will help patients and clinicians with diagnosis and ultimately ensure that patients with nGD are able to access new medicines and the right clinical care. View
Scientific papers A charitable access program for patients with lysosomal storage disorders in underserved communities worldwide Patients in some countries face challenges accessing treatments owing to low healthcare provider (HCP) awareness and education regarding rare diseases View
Scientific papers Challenges in Transition From Childhood to Adulthood Care in Rare Metabolic Diseases Results From the First Multi-Center European Survey Download
IGA materials IGA Impact Report (2020) IGA's Impact Report for 2020 Download
Video Webinar: Wellness for GD patients webinar Dr Seema Kanwall joined our webinar to talk about maximising your wellbeing. Watch