Promising new drug targets rare brain disorder in phase 3 trial
NCT ID NCT04849741
First seen Dec 10, 2025 · Last updated May 23, 2026 · Updated 23 times
Summary
This study tests an experimental drug called zilganersen (ION373) in 54 people with Alexander disease, a rare brain disorder. The goal is to see if the drug can improve or stabilize walking and other motor functions. Participants receive the drug or a placebo via spinal injection, and the study is double-blind, meaning neither they nor their doctors know who gets which.
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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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Amsterdam Universitair Medische Centra - Academisch Medisch Centrum
Amsterdam, North Holland, 1105 AZ, Netherlands
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Children's Hospital of Atlanta
Atlanta, Georgia, 30329, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH, United Kingdom
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Lucile Packard Children's Hospital Stanford
Palo Alto, California, 94304, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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McGill University Health Centre
Montreal, Quebec, H3A 0G4, Canada
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Murdoch Children's Research Institute
Parkville, Victoria, 3052, Australia
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National Center of Neurology and Psychiatry
Tokyo, Kodaira-shi, 187-8551, Japan
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Ospedale Pediatrico Bambino Gesù
Roma, 165, Italy
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Ospedale dei Bambini Vittore Buzzi
Milan, 20154, Italy
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Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906, Israel
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University College London Hospitals NHS Foundation Trust
London, WC1N 3BG, United Kingdom
Conditions
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