Scientists launch major study to map devastating childhood disease
NCT ID NCT07432880
Summary
This study aims to better understand how a severe, rare metabolic disorder called Methylmalonic Acidemia (MMA) progresses in children. Researchers will follow up to 30 children aged 16 or younger, including some who have had liver transplants, to track their health and identify reliable signs of disease activity in their blood. The information gathered will be used to help design future treatment trials, but this study itself does not test any new therapies.
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Contacts and locations
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Study contacts
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CHOP (Children's hospital of Philadelphia)
Philadelphia, Pennsylvania, 19104, United States
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GOSH NHS (Great Ormond Street Hospital for Children)
London, WC1N 3JH, United Kingdom
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Hospital Universitario 12 de Octubre
Madrid, 28041, Spain
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OBGP (Bambino Gesu Ospedale Pediatrico)
Roma, 00146, Italy
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OSR_San Raffaele
Milan, 20132, Italy
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Royal Manchester Children Hospital
Manchester, M13 9WL, United Kingdom
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SJD_San Joan de Deù Children's Hospital
Barcelona, 08950, Spain
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UPMC (Children's hospital of Pittsburgh)
Pittsburgh, Pennsylvania, 15224, United States
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Conditions
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