Gaucher disease: who works on it

One card per institution. Filter by target, by country, or show only institutions in EU-13 (Horizon Europe widening) countries.

• All targets
• GBA1 5
• LRRK2 1
• MEK 0
• NRF2 0
• SNCA 0

EU-13 is a filter, not a ranking: it only narrows the list.

24 of 83 institutions, ordered by recent publications and open trials. This is not a ranking of quality.

Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

• Terminated

  A Study of Home vs Hospital Treatment in People With Fabry, Gaucher or Hunter Conditions in Mexico

  NCT07494058· Takeda· Updated 11 Sept 2026

  “Study was terminated early due to structural and methodological limitations identified in the primary data sources impacting validity, completeness, and clinical robustness of the expected results”Registry text, quoted unchanged. Source

• Withdrawn

  An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3

  NCT05815004· AVROBIO· Updated 9 Aug 2023

  “Further development of programs halted”Registry text, quoted unchanged. Source

Trials whose registry record says individual participant data (IPD) will be shared.

• The Efficacy and Safety of Venglustat Compared With Cerezyme in Pediatric Participants 2 to 11 Years of Age With Gaucher Disease Type 3 (GD3)

  NCT07927140· Sanofi· Source

• Parkinson's Disease in Type 1 Gaucher Disease

  NCT07886879· Icahn School of Medicine at Mount Sinai· Source

• Genetic Studies of Lysosomal Storage Disorders

  NCT00001215· National Human Genome Research Institute (NHGRI)· Source

• A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion

  NCT05669729· Takeda· Source

• Study of Velaglucerase Beta ERT in Children With Gaucher Disease

  NCT07847411· CANbridge Life Sciences Ltd.· Source

• A Study of Home vs Hospital Treatment in People With Fabry, Gaucher or Hunter Conditions in Mexico

  NCT07494058· Takeda· Source

• Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

  NCT05222906· Sanofi· Source

• Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

  NCT02843035· Genzyme, a Sanofi Company· Source

• Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

  NCT03485677· Sanofi· Source

• Survey Study for Velaglucerase Alfa (VPRIV) in Japan

  NCT03625882· Takeda· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

• Organoids 2
• CRISPR-Cas Systems 1
• Proteomics 1
• Metabolomics 1
• Magnetic Resonance Imaging 3
• Cohort Studies 1
• Genome-Wide Association Study 2

• HEPES in Cell Culture Alters the Multi-Omics Profile Exhibited by Gaucher Disease Fibroblasts.

  J Cell Biochem, Jan 2026

See which targets of this disease your abstract mentions, and go to the people working on them.