Could a daily injection spare babies with dwarfism from risky neck surgery?
NCT ID NCT04554940
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a drug called vosoritide in infants with achondroplasia (the most common form of dwarfism) who are at high risk of needing a surgery to relieve pressure on the spinal cord at the base of the skull. The main goal is to see if the drug is safe and if it can help enlarge the skull opening, potentially reducing the need for surgery. About 20 babies under 12 months old will receive the drug or standard care, and their health will be closely monitored.
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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