Rare disease drug safety tracked in tiny french study
NCT ID NCT06949358
First seen Apr 14, 2026 · Last updated Jun 16, 2026 · Updated 13 times
Summary
This study followed 3 people with acid sphingomyelinase deficiency (ASMD) who had already completed earlier trials of the enzyme replacement therapy olipudase alfa. The goal was to keep an eye on safety and side effects while providing the drug until it was officially covered in France. Participants received infusions every two weeks, and researchers tracked any medical issues that came up.
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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
Paris, 75020, France
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Investigational Site Number : 2500002
Bron, 69500, France
Conditions
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