New mRNA therapy targets rare sugar-storage disease
NCT ID NCT05095727
First seen Jan 05, 2026 · Last updated Apr 28, 2026 · Updated 20 times
Summary
This early-stage study tests an experimental mRNA medicine called mRNA-3745 in 15 people with glycogen storage disease type 1a (GSD1a), a rare genetic condition that causes dangerously low blood sugar. The treatment aims to help the body produce a missing enzyme to better control blood sugar levels. The main goal is to check safety and how well the body handles the drug.
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This is a summary of
the original study
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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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AP-HP - Hôpital Antoine Béclère
Clamart, 92140, France
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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CHRU Tours - Hopital Clocheville
Tours, 37000, France
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Duke University Medical Center
Durham, North Carolina, 27713, United States
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Hospital Regional Universitario de Malaga
Málaga, 29011, Spain
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Hospital Universitario 12 de Octubre
Madrid, 28026, Spain
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Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04-730, Poland
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Stollery Children's Hospital University of Alberta
Edmonton, Alberta, T6G 2R7, Canada
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The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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The University of Texas Health Science Center at Houston
Houston, Texas, 77030-1501, United States
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Universitair Medisch Centrum Groningen
Groningen, 9713 GZ, Netherlands
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University of Connecticut Health Center
Farmington, Connecticut, 06030-0001, United States
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University of Utah
Salt Lake City, Utah, 84132-0001, United States
Conditions
Explore the condition pages connected to this study.