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Recruiting trial = Recruiting trial
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National clinical trial(s)

FRANCE

AUVERGNE-RHONE-ALPES
BRON

A study of the safety, efficacy, pharmacokinetics and pharmacodynamics of recombinant human alpha glucosidase (rhGAA) treatment in patients >6 m and <36 m old with infantile onset Pompe disease (Phase II) - Terminated -
CHU de Lyon HCL - GH Est-Hôpital Femme Mère Enfant
Service d'endocrinologie et de diabétologie pédiatriques et maladies héréditaires du métabolisme

FRANCE

AUVERGNE-RHONE-ALPES
BRON

A dose-ranging study of the safety, efficacy, pharmacokinetics and phamacodynamics of recombinant human Acid Alpha Glucosidase (rhGAA) treatment in patients <6 m old with infantile onset Pompe disease (Phase II) - Terminated -
CHU de Lyon HCL - GH Est-Hôpital Femme Mère Enfant
Service d'endocrinologie et de diabétologie pédiatriques et maladies héréditaires du métabolisme

FRANCE

ILE-DE-FRANCE
PARIS

Observational Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme
CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
Groupement hospitalier universitaire Pitié-Salpétrière

ITALY

CAMPANIA
NAPOLI

Funded by an IRDiRC memberCombination therapy of enzyme stimulation (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease
Azienda Ospedaliera Universitaria "Federico II"
Laboratorio Dipartimento di Pediatria

NETHERLANDS

Zuid-Holland
ROTTERDAM

Single-Center, Open-Label Study of Safety, Pharmacokinetics and Efficacy of rhGAA in Patients With Late-Onset Pompe Disease
Erasmus MC - Erasmus Medisch Centrum
Centrum voor Lysosomale en Metabole Ziekten

UNITED KINGDOM

Greater Manchester
ADDRESS: NOT PROVIDED - UK

UNITED KINGDOM

Greater London
LONDON

UNITED KINGDOM

Greater Manchester
SALFORD

Multinational clinical trial(s)