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Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.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

• A phase 2, randomized, double-blind study of S-606001 as an add-on to enzyme replacement therapy in patients with late-onset Pompe disease.

  2025-522146-40-00· Shionogi B.V.· Source

• Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

  NCT05164055· Genzyme, a Sanofi Company· Source

• A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

  NCT04848779· Sanofi· Source

• Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

  NCT04910776· Sanofi· Source

• China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

  NCT06666413· Genzyme, a Sanofi Company· Source

• A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

  NCT03019406· Genzyme, a Sanofi Company· Source

• Avalglucosidase Alfa Pregnancy Study

  NCT05734521· Sanofi· Source

• GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE

  2023-508000-37-01· Rigshospitalet· Source

• Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen

  NCT04676373· Genzyme, a Sanofi Company· Source

• Treatment Frequency Reduction in Pompe Disease

  NCT06575829· Erasmus Medical Center· Source

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

• CRISPR-Cas Systems 1
• Magnetic Resonance Imaging 9
• Cohort Studies 3

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