Experimental gene therapy offers hope for babies with rare brain disease
NCT ID NCT04771416
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 26 times
Summary
This study tests a gene therapy called PBKR03 for children under 9 months with early infantile Krabbe disease, a severe genetic disorder affecting the brain and nerves. The therapy aims to deliver a working copy of the GALC gene to help stop or slow the disease. Researchers will check safety and effectiveness by testing different doses in 24 children.
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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 UMC
Amsterdam, Netherlands
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Ann & Robert Lurie
Chicago, Illinois, 60611, United States
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Hospital De Clinicas De Porto Alegre
Porto Alegre, Brazil
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Manchester University
Manchester, United Kingdom
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Montreal Children's Hospital
Montreal, Canada
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New York-Presbyterian
New York, New York, 10065, United States
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Shaare Zadek Medical Center
Jerusalem, Israel
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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University of Utah School of Medicine
Salt Lake City, Utah, 84132, United States
Conditions
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