Could this drug help babies with dwarfism avoid risky brain surgery?
NCT ID NCT04554940
First seen Mar 13, 2026 · Last updated May 08, 2026 · Updated 5 times
Summary
This study tests a drug called vosoritide in infants with achondroplasia (a common form of dwarfism) who are at high risk of needing brain surgery due to a narrow skull opening. The main goal is to see if the drug is safe and if it can help the skull opening grow larger, potentially reducing the need for surgery. About 20 babies under 12 months old are participating.
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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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Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital
London, SE1 7EH, United Kingdom
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Murdoch Children's Research Institute
Parkville, Victoria, 3052, Australia
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Sheffield Children's NHS Foundation Trust
Sheffield, S10 2TH, United Kingdom
Conditions
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