Old drug offers new hope for kids with devastating muscle disease
NCT ID NCT04007562
Summary
This study aimed to review the safety and effectiveness of hydroxychloroquine for children with a rare, severe genetic disease called Lipin-1 deficiency. The disease causes painful, life-threatening muscle breakdown episodes. Researchers looked back at medical records of children who had already received the drug on a compassionate basis to see if it helped control their symptoms.
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Contacts and locations
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Locations
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Hôpital Necker-Enfants Malades
Paris, 75015, France
Conditions
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