Scientists track rare disease for clues to better care
NCT ID NCT03204370
Summary
This study aimed to learn more about the long-term progression of a rare, milder form of Morquio A disease in adults. Researchers followed 9 adult patients for 5 years, whether they were on the standard enzyme replacement therapy or not. The goal was to gather detailed information to better understand the disease and find the best ways to measure if treatments are working effectively over time.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for MUCOPOLYSACCHARIDOSIS IV A are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Rheumatology department - Bordeaux University Hospital
Bordeaux, Aquitaine, 33076, France
Conditions
Explore the condition pages connected to this study.