Shaare Zedek Medical Center
Jerusalem, Israel
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Gaucher disease
What is new in Gaucher disease research, which clinical trials are open, and who works on it. From public registries and PubMed, with the source and date of every fact.
Cure status
- FLT201Phase 3 · NCT07223944 · NCT06545136
- imiglucerasePhase 3 · NCT05222906 · NCT02843035
- Velaglucerase betaPhase 3 · NCT07847411
- VenglustatPhase 3 · NCT05222906
- AZ-3102Phase 2/3 · NCT07715084 · NCT07885917
- AlemtuzumabPhase 2 · NCT01962415
- FludarabinePhase 2 · NCT01962415
- HydroxyureaPhase 2 · NCT01962415
- MelphalanPhase 2 · NCT01962415
- ThiotepaPhase 2 · NCT01962415
- venglustat (GZ402671)Phase 2 · NCT02843035
- LY-M001Phase 1/2 · NCT06818838 · NCT06528080
- LY3884961Phase 1/2 · NCT04127578 · NCT04411654 · NCT05487599
- MethylprednisolonePhase 1/2 · NCT04127578 · NCT04411654
- PrednisonePhase 1/2 · NCT04411654
- SirolimusPhase 1/2 · NCT04127578 · NCT04411654
- VGN-R08bPhase 1/2 · NCT07603050 · NCT06272149
- Aldurazyme (laronidase)Phase 1 · NCT04532047
- YH35995Phase 1 · NCT06517914
Closest to patients
- Study of Velaglucerase Beta ERT in Children With Gaucher DiseasePhase 3 trial: Velaglucerase betaCANbridge Life Sciences Ltd. (sponsor) · 1 hospital in ChinaPhase 3Results expected 2028
- A Gaucher Disease Gene Therapy Trial With FLT201Phase 3 trial: FLT201Hospital Universitari Vall d'Hebron · Barcelona, Spain + 31 more hospitalsPhase 3Results expected 2028
- Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal DisordersPhase 2/3 trial: AZ-3102Azafaros B.V. (sponsor)Phase 2/3Results expected 2030
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Latest news
Results, new trials and publications, newest first. Every item links to its source.
Results and new studies, newest first, in plain words.
Plain-language summary pending. Original title shown.
Publication
Insights into morbidity and mortality in Gaucher disease: a cohort study.
Insights into morbidity and mortality in Gaucher disease: a cohort study.
Molecular genetics and metabolism reports
PubMed · PMID 42927298Case report
Extended Deletion in the GBA1 Gene, Masked by Apparent p.Asn409Ser Homozygous: Diagnostic Implications and the Need for an Integrated Genetic Approach.
Extended Deletion in the GBA1 Gene, Masked by Apparent p.Asn409Ser Homozygous: Diagnostic Implications and the Need for an Integrated Genetic Approach.
International journal of molecular sciences
PubMed · PMID 42941894Publication
A multiscale signaling-biophysical framework reveals mechanisms of macrophage-mediated RBC clearance in sickle cell and Gaucher disease.
A multiscale signaling-biophysical framework reveals mechanisms of macrophage-mediated RBC clearance in sickle cell and Gaucher disease.
PNAS nexus
PubMed · PMID 42936285Review
Lysosomal alkalinization: a convergent mechanism in lysosomal storage disorders and classical neurodegenerative diseases.
Lysosomal alkalinization: a convergent mechanism in lysosomal storage disorders and classical neurodegenerative diseases.
Cell communication and signaling : CCS
PubMed · PMID 42983435
Organisations that can help
Helplines, patient groups and support services for you and your family.
Select your country to see the organisations there, starting with the ones closest to you.
Umbrella organisations (they connect national groups)
EURORDIS – Rare Diseases Europe
Alliance of over 1,000 rare disease patient organisations, with a list of rare disease helplines by country.
Visit site: EURORDIS – Rare Diseases Europe
International Gaucher Alliance
International voice for Gaucher patient groups: disease information, support for newly diagnosed, help with charitable treatment access, webinars, advocacy training, International Gaucher Day.
Visit site: International Gaucher Alliance
General support
For anyone who is ill, whatever the disease: patients' rights and legal aid, crisis lines, hospice and palliative care, care in another EU country, and patient organisations' umbrella bodies.
International
European Patients' Forum (EPF)
Umbrella of European patient organisations and national patient coalitions.
Visit site: European Patients' Forum (EPF)
International Alliance of Patients' Organizations
Global alliance of patient organisations of all diseases, promoting patient-centred healthcare.
Visit site: International Alliance of Patients' Organizations
Frontlight is not affiliated with these organisations; we list them because they help.
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Find fellow researchers
Groups with active Gaucher disease work.
Bikur Cholim Hospital
Jerusalem, Israel
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Yale University
New Haven, United States
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University College London
London, United Kingdom
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Hebrew University of Jerusalem
Jerusalem, Israel
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Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, United States
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National Institutes of Health
Bethesda, United States
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Inserm
Paris, France
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Université Paris Cité
Paris, France
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Sanofi (United States)
Bridgewater, United States
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Icahn School of Medicine at Mount Sinai
New York, United States
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Assistance Publique – Hôpitaux de Paris
Paris, France
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