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Pompe Disease
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Pompe Disease trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Pompe Disease trials you may qualify forThis is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult p…
The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an…
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to trac…
This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woma…
This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the d…
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dise…
Study ABX1100-1001 is a first-in-human (FIH), phase 1 study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of a single ascending dose…
Open-label, ascending dose trial of ACTUS-101 administered intravenously.
Pompe disease is a genetic condition which causes muscle weakness over time. People with Pompe disease have a faulty gene that makes an enzyme called acid alpha…