French study tracks Long-Term effects of rare muscle disease treatment
NCT ID NCT05164055
First seen Nov 01, 2025 · Last updated Apr 18, 2026 · Updated 25 times
Summary
This study continues to provide and monitor an enzyme replacement therapy called avalglucosidase alfa for 17 patients in France with Pompe disease, a rare genetic disorder that weakens muscles. The purpose is to collect long-term safety data and see how well the treatment continues to work over approximately four years. Participants had already received this therapy in previous studies and will continue receiving it every two weeks.
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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.