New study aims to unlock clues for rare childhood disease
NCT ID NCT07432880
First seen Feb 27, 2026 · Last updated May 20, 2026 · Updated 11 times
Summary
This study follows up to 30 children (up to age 16) with a rare genetic condition called methylmalonic acidemia (MMA). The goal is to track how the disease progresses over time by looking at blood tests and other health signs. No new treatments are tested here; instead, the information gathered will help design future therapies. Some children who have had a liver transplant are also included.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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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
Explore the condition pages connected to this study.