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7 Resultado(s)

Ensaio em fase de recrutamento = Ensaio em fase de recrutamento; Ensaio a decorrer = Ensaio a decorrer; Financiado por um membro do IRDiRC = ; Membro de uma ERN =

Ensaio (s) clínico (s) nacional (is)

FRANCA

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

FRANCA

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

FRANCA

ILE-DE-FRANCE
PARIS

Observational Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme
APHP.Sorbonne Université, Hôpital de la Pitié-Salpêtrière
Groupement hospitalier universitaire Pitié-Salpétrière

PAISES BAIXOS

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