Scientists track rare disease for clues to better care
Knowledge-focused
Completed
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 informatio…
Sponsor: GOIZET • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:42 UTC