Pompe disease patients get continued access to enzyme therapy in french study
NCT ID NCT05164055
First seen Nov 01, 2025 · Last updated May 21, 2026 · Updated 28 times
Summary
This study gives Pompe disease patients in France continued access to the enzyme replacement therapy avalglucosidase alfa after they finished earlier clinical trials. It monitors long-term safety and how well the treatment works over time. About 17 adults and children with late-onset or infantile-onset Pompe disease will receive the drug every two weeks until it is reimbursed in France or until September 2026.
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Investigational Site Number : 2500001
Marseille, 13385, France
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Investigational Site Number : 2500002
Paris, 75013, France
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Investigational Site Number : 2500003
Lyon, 69003, France
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Investigational Site Number : 2500004
Bordeaux, 33000, France
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Investigational Site Number : 2500005
Brest, 29609, France
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Investigational Site Number : 2500006
Nantes, 44093, France
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Investigational Site Number : 2500007
Nice, 06200, France
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Investigational Site Number : 2500008
Clermont-Ferrand, 63000, France
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Investigational Site Number : 2500009
Lille, 59037, France
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Investigational Site Number : 2500010
Paris, 75015, France
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Investigational Site Number : 2500011
Tours, 37044, France
Conditions
Explore the condition pages connected to this study.