Promising new treatment for rare PMM2-CDG disease enters final testing
NCT ID NCT06892288
First seen Nov 01, 2025 · Last updated Apr 25, 2026 · Updated 29 times
Summary
This study tests a new drug called GLM101 for people with PMM2-CDG, a rare inherited condition that causes problems with movement, balance, and coordination. About 50 children and adults will receive weekly infusions of either GLM101 or a placebo for 24 weeks, followed by 24 weeks where everyone gets the real drug. The goal is to see if GLM101 can improve symptoms like unsteady walking and clumsiness.
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This is a summary of
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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 Hopital Necker-Enfants Malades
Paris, 75015, France
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Azienda Ospedaliero Universitaria Pisana
Pisa, 56126, Italy
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Azienda Ospedaliero Universitaria Policlinico G. Rodolico-San Marco
Catania, 95124, Italy
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Birmingham Women's and Children's NHS Foundation Trust
Birmingham, B4 6NH, United Kingdom
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Hospital Sant Joan de Déu
Esplugues de Llobregat, 08950, Spain
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Hospital Universitario 12 de Octubre
Madrid, 28041, Spain
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Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
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Instytut Matki i Dziecka
Warsaw, 01-211, Poland
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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UZ Leuven, Campus Gasthuisberg
Leuven, 3000, Belgium
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Unidade Local de Saúde de Santo António
Porto, 4099-001, Portugal
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Universitaetsklinikum Muenster
Münster, 48149, Germany
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
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Vseobecna fakultni nemocnice v Praze
Prague, 2, 128 0, Czechia
Conditions
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