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.

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

24 of 200+ 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Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

No stopped trial with a stated reason in this view.

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

• 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 First-in-Human, Single- and Multiple-Ascending Dose Study of YH35995 in Healthy Adult Male Participants

  NCT06517914· Yuhan Corporation· 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

• Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)

  NCT02583672· University of Minnesota· 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

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