Gene therapy hope for babies with fatal brain disease
NCT ID NCT04411654
First seen Jan 05, 2026 · Last updated May 15, 2026 · Updated 21 times
Summary
This study tests a single-dose gene therapy (LY3884961) in 7 infants with Type 2 Gaucher disease, a severe and life-limiting condition. The treatment aims to correct the genetic defect to slow or stop disease progression. Researchers will monitor safety, side effects, and how well the therapy works over 5 years.
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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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Children's Hospital of Pittsburgh, 4401 Penn Avenue
Pittsburgh, Pennsylvania, 15224, United States
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Lysosomal & Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030, United States
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Manchester Centre for Genomic Medicine, 6th Floor, St Mary's Hospital, Oxford Road
Manchester, M13 9WL, United Kingdom
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UCSF Benioff Children's Hospital, 747 52nd St
Oakland, California, 94609, United States
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University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue
Minneapolis, Minnesota, 55454, United States
Conditions
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