MRNA therapy for rare metabolic disease shows early promise but study halted
NCT ID NCT04899310
First seen Jan 05, 2026 · Last updated May 17, 2026 · Updated 22 times
Summary
This study tested an experimental mRNA medicine called mRNA-3705 in 18 people with a rare genetic condition called methylmalonic acidemia (MMA), which causes harmful acid buildup in the body. The goal was to see if the treatment is safe and can lower MMA levels. The study was terminated early, so results are limited.
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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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Locations
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Erasmus MC
Rotterdam, 3015 AA, Netherlands
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Hospital For Sick Children
Toronto, Ontario, M5G 1X8, Canada
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Hospital Universitario 12 de Octubre
Madrid, 28041, Spain
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Hospital Universitario Cruces
Barakaldo, 48903, Spain
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Hôpital Necker - Enfants Malades
Paris, 75015, France
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Lucile Packard Children's Hospital at Stanford
Palo Alto, California, 94304, United States
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Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
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Stollery Children's Hospital University of Alberta
Edmonton, Alberta, T6G 2R7, Canada
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
Conditions
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