Promising treatment study for rare muscle disease never gets off ground
NCT ID NCT04007562
First seen Sep 30, 2025 · Last updated Apr 29, 2026 · Updated 23 times
Summary
This study aimed to look back at the safety and effectiveness of hydroxychloroquine in children with Lipin-1 deficiency, a rare genetic condition causing severe muscle breakdown and pain. Researchers planned to review medical records of patients who had already received the drug on a compassionate basis. However, the study was withdrawn before enrolling any participants, so no new results are available.
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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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Locations
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Hôpital Necker-Enfants Malades
Paris, 75015, France
Conditions
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