New study aims to decode rare bone disease without surgery
NCT ID NCT05284006
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study is for children with Morquio A, a rare genetic disease that affects bones and breathing. Researchers will use safe, non-invasive tests like MRI, X-rays, hearing tests, and walking analysis to track how the disease changes over time. The goal is to better understand the disease and find better ways to measure it in future treatment studies.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
What this could lead to
If successful, this could lead to better ways to track Morquio A progression and define clear endpoints for future treatment trials.
What could go wrong
This is an observational study, not a treatment trial. It will not directly improve symptoms or cure the disease. Results may take years to influence clinical care.
Disclaimer
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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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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Nemours Children's Health, Delaware Valley
RECRUITINGWilmington, Delaware, 19803, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••