New drug shows promise for rare childhood disorder
NCT ID NCT05549219
First seen Jan 07, 2026 · Last updated Apr 24, 2026 · Updated 22 times
Summary
This study tested an experimental drug called GLM101 in 27 people with PMM2-CDG, a rare genetic condition that affects movement and development. Participants received different doses of the drug intravenously over 24 weeks. The main goal was to see if the drug improves coordination and to check its safety.
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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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Clinical Research of West Florida
Tampa, Florida, 33606, United States
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Great Ormond Street Hospital
London, WC1N3JH, United Kingdom
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Hospital Sant Joan de Déu
Barcelona, 08950, Spain
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Mayo Clinic
Rochester, Minnesota, 55905, United States
Conditions
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