Scientists track rare childhood disease to pave way for future cures
NCT ID NCT07432880
Summary
This study aims to better understand how a rare and severe genetic disorder called Methylmalonic Acidemia (MMA) progresses in children. Researchers will follow up to 30 children, aged 16 or younger, who have a severe form of the disease, with or without a liver transplant. The goal is to identify reliable signs and blood test results that show how active the disease is, which will help guide the development of future treatments.
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Contacts and locations
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Study contacts
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Contact
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Locations
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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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