Promising treatment for rare genetic disease continues testing
NCT ID NCT06657859
First seen Mar 04, 2026 · Last updated Apr 24, 2026 · Updated 9 times
Summary
This study offers continued access to the experimental drug GLM101 for people with PMM2-CDG, a rare genetic disorder that affects movement and development. Participants who completed a previous GLM101 trial will receive weekly infusions to see how safe the drug is over the long term and whether it helps with coordination and balance. The goal is to better understand the drug's lasting effects.
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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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Great Ormond Street Hospital
London, WC1N3JH, United Kingdom
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Hospital Sant Joan de Déu
Barcelona, Spain, 08950, Spain
Conditions
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